Orphan drugs in the USA Orphan drugs in the USA| Designation date | Generic name | KEGG | Designation | Trade name | Approved indication | Marketing approval date | Contact company |
| 2016/2/4 | Venetoclax | D10679 | Treatment of acute myeloid leukemia | VENCLEXTA | For the treatment of patients with chronic lymphocytic leukemia (CLL) with 17p deletion, as detected by an FDA approved test, who have received at least one prior therapy | 2016/4/11 | AbbVie Inc. |
| 2015/11/20 | Necitumumab | D10018 | Treatment of squamous non-small cell lung cancer | PORTRAZZA | Use in combination with gemcitabine and cisplatin, for first-line treatment of patients with metastatic, squamous, non-small cell lung cancer. PORTRAZZA is not indicated for treatment of non-squamous, non-small cell lung cancer | 2015/11/24 | Eli Lilly and Company |
| 2015/10/27 | Paricalcitol | D00930 | Treatment of pediatric hyperparathyroidism | ZEMPLAR | Indicated in pediatric patients 10 years and older for the prevention and treatment of secondary hyperparathyroidism associated with Chronic kidney disease (CKD) Stages 3 and 4 and CKD Stage 5 in patients on hemodialysis or peritoneal dialysis | 2016/10/18 | Abbvie, inc. |
| 2015/8/3 | Afatinib | D09733 | Treatment of non-small cell lung cancer with squamous histology. | GILOTRIF | Treatment of patients with metastatic, squamous, non-small cell lung cancer progressing after platinum-based chemotherapy | 2016/4/15 | |
| 2015/5/13 | Adalimumab | D02597 | Treatment of moderate to severe hidradenitis suppurativa (Hurley stage 2 and Hurley stage 3 disease) | HUMIRA | Treatment of moderate to severe hidradenitis suppurativa | 2015/9/9 | AbbVie, Inc. |
| 2015/4/15 | Obinutuzumab | D09321 | Treatment of follicular lymphoma | GAZYVA | Indicated for the treatment of patients with follicular lymphoma (FL) who relapsed after, or are refractory to, a rituximab-containing regimen | 2016/2/26 | Genentech, Inc. |
| 2015/2/24 | Acetylcysteine effervescent tablets for oral solution | D00221 | Preventing hepatic injury from acetaminophin overdose | CETYLEV | Indicated to prevent or lessen hepatic injury after ingestion of a potentially hepatotoxic quantity of acetaminophen in patients with acute ingestion or from repeated supratherapeutic ingestion | 2016/1/29 | Arbor Pharmaceuticals, LLC |
| 2015/2/24 | Deferasirox | D03669 | Treatment of chronic iron overload in alpha-thalassemia | EXJADE | Treatment of chronic iron overload in patients 10 years of age and older with non-transfusion dependent thalassemia (NTDT) syndromes and with a liver iron concentration (LIC) of at least 5 milligrams of iron per gram of liver dry weight (mg Fe/g dw) and serum ferritin greater than 300 mcg/L. | 2013/1/23 2006/8/28 (EU) |
Novartis Pharmaceuticals Corporation Novartis Europharm Limited (EU) |
| 2014/10/9 | Combination of nivolumab and ipilimumab | Treatment of Stage IIb to Stage IV melanoma | Treatment of patients with BRAF V600 wild-type, unresectable or metastatic melanoma | 2015/9/30 | Bristol-Myers Squibb Company | ||
| 2014/10/9 | Olaratumab | D09939 | Treatment of soft tissue sarcoma | LARTRUVO | In combination with doxorubicin, for the treatment of adult patients with soft tissue sarcoma (STS) with a histologic subtype for which an anthracycline-containing regimen is appropriate and which is not amenable to curative treatment with radiotherapy or surgery. | 2016/10/19 | Eli Lilly and Company |
| 2014/9/17 | Technetium Tc 99m tilmanocept | Use in sentinel lymph node detection (SLN) with a hand-held gamma-counter, with scintigraphic imaging, in patients with cancer of the head and neck | LYMPHOSEEK | Guiding sentinel lymph node biopsy, using a hand-held gamma counter in patients with clinically node negative squamous cell carcinoma of the oral cavity. | 2014/6/13 | Navidea Biopharmaceuticals | |
| 2014/9/13 | Mebendazole | D00368 | Treatment of single or mixed gastrointestinal infestations by Trichuris trichiura (whipworm), Ascaris lumbricoides (large roundworm), and Ancylostoma duodenale and Nectar americanus (hookworm). | VERMOX | Treatment of patients one year of age and older withgastrointestinal infections caused by Ascaris lumbricoides (roundworm) and Trichuris trichiura (whipworm) | 2016/10/19 | Janssen Pharmaceutical Research & Development, LLC |
| 2014/8/26 | Gefitinib | D01977 | Treatment of epidermal growth factor receptor mutation-positive non-small cell lung cancer | IRESSA | For the first-line treatment of patients with metastatic non-small cell lung cancer (NSCLC) whose tumors have epidermal growth factor receptor (EGFR) exon 19 deletions or exon 21 (L858R) substitution mutations as detected by an FDA-approved test | 2015/7/13 | AstraZeneca Pharmaceuticals LP |
| 2014/8/7 | Nivolumab | D10316 | Treatment of Hodgkin lymphoma | OPDIVO | Classical Hodgkin lymphoma that has relapsed or progressed after autologous hematopoietic stem cell transplantation (HSCT) and post-transplantation brentuximab vedotin | 2016/5/17 | Bristol-Myers Squibb Co. |
| 2014/6/30 | Lumacaftor/ivacaftor | D10685 | Treatment of cystic fibrosis | ORKAMBI | Treatment of cystic fibrosis (CF) in patients age 6-11 year old who are homozygous for the F508del mutation in the CFTR gene | 2016/9/28 | Vertex Pharmaceuticals Inc. |
| Treatment of cystic fibrosis in patients age 12 years and older who are homozygous for F508del mutation in the CFTR gene | 2015/7/02 2015/11/19 (EU) |
Vertex Pharmaceuticals Inc. | |||||
| 2014/5/13 | Adalimumab | D02597 | Treatment of non-infectious intermediate, posterior, or pan-uveitis, or chronic non-infectious anterior uveitis | HUMIRA | Indicated for the treatment of non-infectious intermediate, posterior and panuveitis in adult patients | 2016/6/30 | AbbVie, Inc. |
| 2014/2/14 | Phoxilium | For use as a replacement solution in patients undergoing continuous renal replacement therapy | As a replacement solution in Continuous Renal Replacement Therapy (CRRT) and in case of drug poisoning when CRRT is used to remove dialzable substances | 2015/1/13 | Gambro Renal Products, Inc. | ||
| 2014/2/14 | Rosuvastatin | D01915 | For the treatment of pediatric homozygous familial hypercholesterolemia | CRESTOR | An adjunct to diet to reduce LDL-C, Total-C, nonHDL-C and ApoB in children and adolescents 7 to 17 years of age with homozygous familial hypercholesterolemia, either alone or with other lipid-lowering treatments (e.g., LDL apheresis). | 2016/5/27 | iPR Pharmaceuticals, Inc. |
| 2013/12/31 | Gallium dotatate Ga-68 | Diagnostic for the clinical management of neuroendocrine tumors | NETSPOT | For use after radiolabeling with Ga 68, with positron emission tomography (PET) for localization of somatostatin receptor positive neuroendocrine tumors (NETs) in adult and pediatric patients. | 2016/6/1 | Advanced Accelerator Applications, USA | |
| 2013/12/20 | Tacrolimus | D00107 | Prophylaxis of organ rejection in patients receiving allogeneic kidney transplant | ENVARSUS XR | Prophylaxis of organ rejection in kidney transplant patients converted from tacrolimus immediate-release formulations in combination with other immunosuppressants | 2015/7/10 | Veloxis Pharmaceuticals, Inc. |
| 2013/12/5 | Canakinumab | D09315 | Treatment of familial mediterranean fever | ILARIS | Indicated for Familial Mediterranean Fever (FMF) in adult and pediatric patients | 2016/9/23 | Novartis Pharmaceuticals Corporation |
| Treatment of hyperimmunoglobulinemia D and periodic fever syndrome | Indicated for Hyperimmunoglobulin D Syndrome (HIDS)/Mevalonate Kinase Deficiency (MKD) in adult and pediatric patients | ||||||
| 2013/11/26 | Bendamustine hydrochloride | D07085 | Treatment of Follicular Lymphoma, Small Lymphocytic Lymphoma, Lymphoplasmacytic Lymphoma, Splenic Marginal Zone Lymphoma, Extranodal Marginal Zone B-cell Lymphoma of Mucosa-Associated Lymphoma Tissue (MALT), and Nodal Marginal Zone Lymphoma (Collectively Indolent B-cell Non-Hodgkin's Lymphoma) | TREANDA | Treatment of Indolent B-cell non-Hodgkin lymphoma (NHL) that has progressed during or within six months of treatment with rituximab or a rituximab-containing regimen. | 2008/10/31 | Cephalon, Inc. |
| 2013/11/20 | Filgrastim | D03235 | Treatment of subjects at risk of developing myelosuppression after a radiological or nuclear incident | NEUPOGEN | To increase survival in patients acutely exposed to myelosuppressive doses of radiation (Hematopoietic Syndrome of Acute Radiation Syndrome). | 2015/3/30 | Amgen, Inc. |
| 2013/11/08 | Eltrombopag | D03978 | Treatment of aplastic anemia | PROMACTA | Treatment of patients with severe aplastic anemia who have had an insufficient response to immunosuppressive therapy. | 2014/8/26 | Novartis Pharmaceuticals Corp. |
| 2013/10/25 | Isavuconazonium sulfate | D10643 | Treatment of zygomycosis | CRESEMBA | Treatment of invasive mucormycosis in patients 18 years of age and older | 2015/3/6 | Astellas Pharma Global Development Inc. |
| 2013/10/16 | Olaparib | D09730 | Treatment of ovarian cancer | LYNPARZA | Use of as monotherapy for patients with deleterious or suspected deleterious germline BRCA mutated (as detected by an FDA-approved test) advanced ovarian cancer who have been treated with three or more prior lines of chemotherapy | 2014/12/19 2014/12/16 (EU) |
AstraZeneca Pharmaceuticals LP AstraZeneca AB (EU) |
| 2013/10/15 | Ibrutinib | D10223 | Treatment of Waldenstrom's macroglobulinemia | IMBRUVICA | Treatment of patients with Waldenstrom's macroglobulinemia | 2015/1/29 2014/10/21 (EU) |
Pharmacyclics, LLC Janssen-Cilag International NV (EU) |
| 2013/10/15 | Idelalisib | D10560 | Treatment of chronic lymphocytic leukemia and small lymphocytic lymphoma | ZYDELIG | (1) Relapsed chronic lymphocytic leukemia (CLL), in combination with rituximab, in patients for whom rituximab alone would be considered appropriate therapy due to other co-morbidities; and (2) Relapsed small lymphocytic lymphoma (SLL) in patients who have received at least two prior systemic therapies. | 2014/7/23 | Gilead Sciences, Inc. |
| 2013/9/27 | Ceritinib | D10551 | Treatment of patients with non-small cell lung cancer (NSCLC) that is anaplastic lymphoma kinase(ALK)-positive | ZYKADIA | Treatment of patients with anaplastic lymphoma kinase (ALK)-positive metastatic non-small cell lung cancer (NSCLC) who have progressed on or are intolerant to crizotinib. | 2014/4/29 | Novartis Pharmaceuticals Corp. |
| 2013/9/26 | Ethiodized oil injection | D04082 | Management of patients with known hepatocellular carcinoma (HCC) | LIPIODOL | Selective hepatic intra-arterial use for imaging tumors in adults with known hepatocellular carcinoma (HCC) | 2014/4/4 | Guerbet LLC |
| 2013/9/26 | Idelalisib | D10560 | Treatment of follicular lymphoma | ZYDELIG | Relapsed follicular B-cell non-Hodgkin lymphoma (FL) in patients who have received at least two prior systemic therapies. | 2014/7/23 | Gilead Sciences, Inc. |
| 2013/9/19 | Riociguat | D09572 | Treatment of chronic thromboembolic pulmonary hypertension | ADEMPAS | Treatment of adults with persistent/recurrent chronic thromboembolic pulmonary hypertension (CTEPH) WHO Group 4, after surgical treatment, or inoperable CTEPH, to improve exercise capacity and WHO functional class | 2013/10/8 2014/3/27 (EU) |
Bayer HealthCare Pharmaceuticals, Inc. Bayer Pharma AG (EU) |
| 2013/9/19 | Riociguat | D09572 | Treatment of pulmonary arterial hypertension. | ADEMPAS | Treatment of adults with pulmonary arterial hypertension (PAH) WHO Group 1, to improve exercise capacity, WHO functional class and to delay clinical worsening. | 2013/10/08 2014/3/27 (EU) |
Bayer HealthCare Pharmaceuticals, Inc. Bayer Pharma AG (EU) |
| 2013/9/12 | Evolocumab | D10557 | Treatment of homozygous familial hypercholesterolemia | REPATHA | As an adjunct to diet and other LDL-lowering therapies (e.g., statins, ezetimibe, LDL apheresis) in patients with HoFH who require additional lowering of LDL-C. | 2015/8/27 | Amgen Inc. |
| 2013/9/11 | Denosumab | D03684 | Treatment of hypercalcemia in malignancy | XGEVA | To treat hypercalcemia of malignancy refractory to biphosphonate therapy. | 2014/12/5 | Amgen, Inc. |
| 2013/8/16 | Dantrolene sodium suspension for injection | D02274 | Treatment of malignant hyperthermia syndrome | RYANODEX | Treatment of malignant hyperthermia in conjunction with appropriate supportive measures and for the prevention of malignant hyperthermia in patients at high risk. | 2014/7/22 | Eagle Pharmaceuticals, Inc. |
| 2013/8/9 | Uridine triacetate | D09985 | Treatment of hereditary orotic aciduria | Treatment of hereditary orotic aciduria | 2015/9/4 | Wellstat Therapeutics, Inc. | |
| 2013/6/27 | Intravenous carbamazepine | D00252 | Treatment of epilepsy patients who cannot take anything by mouth (NPO) | CARNEXIV | Indication: replacement therapy for oral carbamazepine formulations, when oral administration is temporarily not feasible, in adults with the following seizure types: (1) Partial seizures with complex symptomology; (2) Generalized clonic-tonic seizures; and Mixed seizure patterns which include the above or other partial or generalized seizures(3) | 2016/10/7 | Lundbeck LLC |
| 2013/5/6 | Daratumumab | D10777 | Treatment of multiple myeloma | DARZALEX | DARZALEX in combination with lenalidomide and dexamethasone, or bortezomib and dexamethasone, for the treatment of patients with multiple myeloma who have received at least one prior therapy. | 2016/11/21 | Janssen Biotech, Inc. |
| For the treatment of patients with multiple myeloma who have received at least 3 prior lines of therapy including a proteasome inhibitor and an immunomodulatory agent or are double refractory to a proteasome inhibitor and an immunomodulatory agent | 2015/11/16 | Janssen Research & Development, LLC | |||||
| 2013/5/6 | Isavuconazonium sulfate | D10643 | Treatment of invasive aspergillosis | CRESEMBA | Treatment of invasive aspergillosis in patienst 18 years of age and older | 2015/3/6 | Astellas Pharma Global Development Inc. |
| 2013/4/12 | Anti-inhibitor coagulant complex | Routine prophylaxis to prevent or reduce the frequency of bleeding episodes in hemophilia A and B patients with inhibitors | FEIBA | Routine prophylaxis to prevent or reduce the frequency of bleeding episodes in hemophilia A and B patients with inhibitors | 2013/12/16 | Baxalta US, Inc. | |
| 2013/01/30 | Enalapril maleate (powder for oral solution) | D00621 | Treatment of hypertension in pediatric patients | EPANED | Treatment of hypertension in adults and children older than one month, to lower blood pressure. | 2013/8/13 | Silvergate Pharmaceuticals, Inc. |
| 2013/1/23 | Nivolumab | D10316 | Treatment of Stage IIb to IV melanoma | OPDIVO | Treatment of patients with unresectable or metastatic melanoma and disease progression following ipilimumab and, if BRAF V600 mutation positive, a BRAF inhibitor | 2014/12/22 | Bristol-Myers Squibb Co. |
| 2012/12/27 | Lenvatinib | D09920 | Treatment of follicullar, medullary, anaplastic, and metastatic or locally advanced papillary thyroid cancer | LENVIMA | Treatment of patients with locally recurrent or metastatic, progressive, radioactive iodine refractory differentiated thyroid cancer | 2015/2/13 2015/5/28 (EU) |
Eisai, Inc. Eisai Europe Ltd (EU) |
| 2012/12/27 | Prothrombin complex concentrate (human) | Treatment of patients needing urgent reversal of Vitamin K antagonist therapy for treatment of major bleeding and/or surgical procedures | KCENTRA | Urgent reversal of acquired coagulation factor deficiency induced by vitamin K antagonist therapy (VKA, e.g., warfarin) in adult patients with the need for urgent surgery/invasive procedure. | 2013/12/13 | CSL Behring | |
| 2012/12/27 | Prothrombin complex concentrate (human) | Treatment of patients needing urgent reversal of Vitamin K antagonist therapy for treatment of major bleeding and/or surgical procedures | ADEMPAS | Urgent reversal of acquired coagulation factor deficiency induced by Vitamin K antagonist (VKA, e.g., warfarin) therapy in adult patients with acute major bleeding. | 2013/4/29 | CSL Behring | |
| 2012/12/8 | Methylene blue | D02312 | Treatment of hereditary and acquired methemoglobinemia | PROVAYBLUE | For treatment of pediatric and adult patients with acquired methemoglobinemia. | 2016/4/8 | Provepharm SAS |
| 2012/12/3 | Afatinib | D09733 | Treatment of epidermal growth factor receptor (EGFR) mutation-positive non-small cell lung cancer (NSCLC). | GILOTRIF | First-line treatment of patients with metastatic non-small cell lung cancer (NSCLS) whose tumors have epidermal growth factor receptor (EGFR) exon 19 deletions or exon 21 (L858R) substitution mutations as detected by an FDA-approved test. | 2013/7/12 | Boehringer Ingelheim Pharmaceuticals, Inc. |
| 2012/12/3 | Ibrutinib | D10223 | Treatment of mantle cell lymphoma | IMBRUVICA | Treatment of patients with mantle cell lymphoma (MCL) who have received at least one prior therapy | 2013/11/13 | Pharmacyclics, LLC |
| 2012/11/19 | Pembrolizumab | D10574 | Treatment of Stage IIB through IV malignant melanoma | KEYTRUDA | Treatment of patients with unresectable or metastatic melanoma and disease progression following ipilimumab and, if BRAF V600 mutation positive, a BRAF inhibitor. | 2014/9/4 | Merck, Sharp & Dohme Corp. |
| 2012/10/31 | Coagulation factor IX (recombinant) | D08797 | Prophylactic use to prevent or reduce the frequency of bleeding episodes in patients with hemophilia B (routine prophylaxis in patients where there is no evidence or suspicion of bleeding) | RIXUBIS | Adults with Hemophilia B for routine prophylaxis to prevent or reduce the frequency of bleeding episodes. | 2013/6/26 | Baxalta US, Inc. |
| 2012/10/31 | Sirolimus | D00753 | Treatment of lymphangioleiomyomatosis | RAPAMUNE | Treatment of lymphangioleiomyomatosis (LAM) | 2015/5/28 | Pfizer, Inc. |
| 2012/9/25 | Japanese encephalitis vaccine, inactivated, adsorbed | Prevention of Japanese encephalitis virus in pediatric patients. | IXIARO | To extend the age range to include infants, children, and adolescents 2 months to less than 17 years of age for active immunization for the prevention of disease caused by Japanese encephalitis virus. | 2013/5/17 | Intercell AG | |
| 2012/9/20 | Trametinib and dabrafenib | Treatment of Stage IIb through IV melanoma. | MEKINIST AND TAFINLAR | TAFINLAR (dabrafenib) in combination with trametinib for treatment of patients with unresectable or metastatic melanoma with BRAF V600E or V600K mutations as detected by an FDA-approved test. This indication is based on the demonstration of durable response rate. Improvement in disease-related symptoms or overall survival has not been demonstrated for TAFINLAR in combination with trametinib. | 2014/1/9 | Novartis Pharmaceuticals Corp. | |
| 2012/9/20 | Trametinib and dabrafenib | Treatment of Stage IIb through IV melanoma. | MEKINIST AND TAFINLAR | MEKINIST (trametinib) in combination with dabrafenib for treatment of patients with unresectable or metastatic melanoma with BRAF V600E or V600K mutations as detected by an FDA-approved test. This indication is based on the demonstration of durable response rate. Improvement in disease-related symptoms or overall survival has not been demonstrated for MEKINIST in combination with dabrafenib. | 2014/1/8 | Novartis Pharmaceuticals Corp. | |
| 2012/9/20 | Venetoclax | D10679 | Treatment of chronic lymphocytic leukemia | VENCLEXTA | Treatment of patients with chronic lymphocytic leukemia (CLL) with 17p deletion, as detected by an FDA approved test, who have received at least one prior therapy | 2016/4/11 | AbbVie, Inc |
| 2012/9/4 | Canakinumab | D09315 | Treatment of TNF-receptor associated periodic syndrome (TRAPS) | ILARIS | Indicated for Tumor Necrosis Factor Receptor Associated Periodic Syndrome (TRAPS) in adult and pediatric patients | 2016/9/23 | Novartis Pharmaceuticals Corporation |
| 2012/8/20 | Mercaptopurine oral solution | D00161 | Treatment of acute lymphoblastic leukemia in pediatric patients | PURIXAN XALUPRINE (EU) |
Treatment of patients with acute lymphoblastic leukemia as part of a combination regimen. | 2014/4/28 2012/3/9 (EU) |
Nova Laboratories Limited Nova Laboratories Ltd (EU) |
| 2012/8/20 | Panobinostat | D10019 | Treatment of multiple myeloma | FARYDAK | Treatment of patients with multiple myeloma who have received at least 2 prior regimens, including bortezomib and an immunomodulatory agent | 2015/2/23 2015/8/28 (EU) |
Novartis Pharmaceuticals Corporation Novartis Europharm Ltd (EU) |
| 2012/7/31 | Rucaparib | D10079 | Treatment of ovarian cancer | RUBRACA | As monotherapy for the treatment of patients with deleterious BRCA mutation (germline and/or somatic) associated advanced ovarian cancer who have been treated with two or more chemotherapies | 2016/12/19 | Clovis Oncology, Inc. |
| 2012/7/31 | Tocilizumab | D02596 | Treatment of pediatric patients (age 16 years and younger) with polyarticular-course juvenile idiopathic arthritis | ACTEMRA | Treatment of active polyarticular juvenile idiopathic arthritis in patients 2 through 16 years of age. | 2013/4/29 | Genentech, Inc. |
| 2012/5/30 | Bortezomib | D03150 | Treatment of mantle cell lymphoma. | VELCADE | Treatment of patients with mantle cell lymphoma who have received at least 1 prior therapy. | 2006/12/8 | Millennium Pharmaceuticals, Inc. |
| 2012/5/30 | Bortezomib | D03150 | Treatment of mantle cell lymphoma. | VELCADE | Treatment of patients with mantle cell lymphoma | 2014/10/8 | Millennium Pharmaceuticals, Inc. |
| 2012/5/14 | Eribulin mesylate | D08914 | Treatment of advanced soft tissue sarcoma | HALAVEN | Treatment of patients with unresectable or metastatic liposarcoma who have received a prior anthracyclne-containing regimen | 2016/1/28 | Eisai Inc. |
| 2012/4/27 | Recombinant fusion protein linking coagulation factor IX with albumin (rIX-FP) | Treatment of patients with congenital factor IX deficiency (hemophilia B) | IDELVION | Indicated for the (1) on-demand control and prevention of bleeding episodes, (2) perioperative management of bleeding, and (3) routine prophylaxis to prevent or reduce the frequency of bleeding episodes. | 2016/3/4 | CSL Behring, LLC | |
| 2012/4/6 | Ibrutinib | D10223 | Treatment of chronic lymphocytic leukemia (CLL) | IMBRUVICA | Indicated for the treatment of patients with chronic lymphocytic leukemia without 17p deletion who have not received at least one prior therapy (first line therapy). | 2016/3/4 | Pharmacyclics, LLC |
| Treatment of patients with chronic lymphocytic leukemia with 17p deletion who have not received at least one prior therapy | 2014/7/28 | Pharmacyclics, LLC | |||||
| Treatment of patients with chronic lymphocytic leukemia (CLL) who have received at least one prior therapy | 2014/2/12 | Pharmacyclics, LLC | |||||
| 2012/2/17 | Obinutuzumab | D09321 | Treatment of chronic lymphocytic leukemia | GAZYVA GAZYVARO (EU) |
Treatment of patients with previously untreated chronic lymphocytic leukemia in combination with chlorambucil. | 2013/11/1 2014/7/23 (EU) |
Genentech, Inc. Roche Registration Ltd (EU) |
| 2012/2/16 | Ramucirumab | D09371 | Treatment of gastric cancer | CYRAMZA | Treatment of advanced gastric cancer or gastro-esophageal junction adenocarcinoma, as a single-agent after prior fluoropyrimidine-or platinum-containing therapy. | 2014/4/21 | Eli Lilly and Company |
| 2012/2/16 | Ramucirumab | D09371 | Treatment of gastric cancer | CYRAMZA | Treatment of advanced gastric or gastro-esophageal junction adenocarcinoma, as a single agent or in combination with paclitaxel, after prior fluoropyrimidine- or platinium-containing chemotherapy. | 2014/11/5 | Eli Lilly and Company |
| 2011/12/12 | Sorafenib | D06272 | Treatment of medullary thyroid cancer, anaplastic thyroid cancer, and recurrent or metastatic follicular or papillary thyroid cancer | NEXAVAR | Treatment of patients with locally recurrent or metastatic, progressive, differentiated thyroid carcinoma (DCT) that is refractory to radioactive iodine treatment. | 2013/11/22 2006/7/19 (EU) |
Bayer HealthCare Pharmaceuticals, Inc. Bayer Pharma AG (EU) |
| 2011/12/21 | Riboflavin ophthalmic solution ultraviolet-A (UVA) irradiation | Treatment of corneal ectasia following refractive surgery | PHOTREXA VISCOUS | Treatment of corneal ectasia following refractive surgery | 2016/7/15 | Avedro, Inc. | |
| 2011/9/16 | Nimodipine | D00438 | Treatment of subarachnoid hemorrhage. | NYMALIZE | Improvement of neurological outcome by reducing the incidence and severity of ischemic deficits in adult patients with subarachnoid hemorrhage (SAH) from ruptured intracranial berry aneurysms regardless of their post-ictus neurological condition (i.e., Hunt and Hess Grades I-V). | 2013/5/10 | Arbor Pharmaceuticals, Inc. |
| 2011/9/2 | Riboflavin ophthalmic solution & ultraviolet A | Treatment of keratoconus | PHOTREXA VISCOUS | Treatment of progressive keratoconus | 2016/4/15 | ||
| 2011/8/25 | Lanreotide acetate | D04666 | Treatment of neuroendocrine tumors | SOMATULINE | Treatment of patients with unresectable,well- or moderately-differentiated locally advanced or metastatic gastroenteropancreatic neuroendocrine tumors to improve progression-free survival | 2014/12/16 | Ipsen Biopharmaceuticals, Inc. |
| 2011/6/29 | Botulism antitoxin heptavalent (A, B, C, D, E, F, G) (Equine) | Treatment of botulism. | Treatment of symptomatic botulism following documented or suspected exposure to botulinum neurotoxin serotypes A, B, C, D, E, F, or G in adults and pediatric patients | 2013/3/22 | Cangene Corporation | ||
| 2011/6/29 | Nintedanib | D10396 | Treatment of patients with idiopathic pulmonary fibrosis. | OFEV | Treatment of idiopathic pulmonary fibrosis | 2014/10/15 | Boehringer Ingelheim Pharmaceuticals, Inc. |
| 2011/6/7 | Gabapentin enacarbil | D09539 | Treatment of postherpetic neuralgia | HORIZANT | Management of postherpetic neuralgia in adults. | 2012/6/6 | XenoPort, Inc. |
| 2011/4/29 | Immune globulin intraveous (human) | Treatment of idiopathic thrombocytopenic purpura (also known as primary immune thrombocytopenia) | GAMMAPLEX | Idiopathic thrombocytopenic purpura | 2013/3/8 | Bio Products Laboratory | |
| 2011/4/18 | Nusinersen | D10881 | Treatment of spinal muscular atrophy | SPINRAZA | Treatment of spinal muscular atrophy in pediatric and adult patients | 2016/12/23 | Biogen, Inc |
| 2011/2/18 | Ixazomib | D10131 | Treatment of multiple myeloma | NINLARO | Use in combination with lenalidomide and dexamethasone for the treatment of patients with multiple myeloma who have received at least one prior therapy | 2015/12/9 | Millennium Pharmaceuticals |
| 2011/1/12 | Dabrafenib | D10104 | Treatment BRAF V600 mutation positive Stage IIB through IV melanoma | TAFINLAR | Treatment of patients with unresectable or metastatic melanoma with BRAF V600E mutation as detected by an FDA approved test. | 2013/5/29 | Novartis Pharmaceuticals Corp. |
| 2011/1/12 | Regorafenib | D10137 | Treatment gastrointestinal stromal tumors | STIVARGA | Treatment of patients with locally advanced, unresectable or metastatic gastrointestinal stromal tumor (GIST) who have been previously treated with imatinib mesylate and sunitinib malate. | 2013/2/25 | Bayer HealthCare Pharmaceuticals, Inc. |
| 2010/12/20 | Denosumab | D03684 | Treatment of patients with giant cell tumor of bone | XGEVA | Treatment of adults and skeletally mature adolescents with giant cell tumor of bone that is unresectable or where surgical resection is likely to result in severe morbidity. | 2013/6/13 | Amgen, Inc. |
| 2010/12/20 | Dinutuximab | D10559 | Treatment of neuroblastoma | UNITUXIN (EU) |
For use in combination with granulocyte-macrophage colony-stimulating factor (GM-CSF), interleukin-2 (IL-2) and 13-cis-retinoic acid (RA), for the treatment of pediatric patients with high-risk neuroblastoma who achieve at least a partial response to prior first-line multiagent, multimodality therapy | 2015/3/10 2015/8/14 (EU) |
United Therapeutics Corporation United Therapeutics Europe Ltd (EU) |
| 2010/12/20 | Trametinib | D10176 | Treatment of Stage IIb through Stage IV melanoma | MEKINIST | Treatment of patients with unresectable or metastatic melanoma with BRAF V600E or V600K mutations as detected by an FDA approved test. | 2013/5/29 | Novartis Pharmaceuticals Corp. |
| 2010/12/20 | Vemurafenib | D09996 | Treatment of patients with IIb to Stage IV melanoma positive for the BRAF(v600) mutation | ZELBORAF | Treatment of unresectable or metastatic melanoma with the BRAFV600E mutation as detected by an FDA-approved test. | 2011/8/17 | Hoffmann-La Roche, Inc. |
| 2010/11/29 | Cabozantinib | D10095 D10062 |
Treatment of follicular, medullary and anaplastic thyroid carcinoma and metastatic or locally advanced papillary thyroid cancer. | COMETRIQ | Treatment of progressive, metastatic medullary thyroid cancer (MTC). | 2012/11/29 2014/3/21 (EU) |
Exelixis, Inc. TMC Pharma Services Ltd |
| 2010/11/23 | Antihemophilic factor (recombinant), Fc fusion protein | D02958 | Treatment of hemophilia A | ELOCTATE | Treatment of adults and children with Hemophilia A (congenital Factor VIII deficiency) for control and prevention of bleeding episodes, perioperative management, and routine prophylaxis to prevent or reduce the frequency of bleeding episodes. | 2014/6/6 | Biogen Idec Inc. |
| 2010/11/23 | Bevacizumab | D06409 | Treatment of fallopian tube carcinoma | AVASTIN | Either in combination with carboplatin and paclitaxel or in combination with carboplatin and gemcitabine, followed by Avastin as a single agent, is indicated for the treatment of patients with platinum-sensitive recurrent epithelial ovarian, fallopian tube, or primary peritoneal cancer. | 2016/12/6 | Genentech, Inc. |
| In combination with paclitaxel, pegylated liposomal doxorubicin, or topotecan for treatment of patients with platinum-resistant, recurrent epithelial ovarian, fallopian tube, or primary peritoneal cancer who have received no more than 2 prior chemotherapy regimens. | 2014/11/14 | ||||||
| 2010/11/8 | Gabapentin | D00332 | Management of postherpetic neuralgia | GRALISE | For the management of postherpetic neuralgia | 2011/1/28 | Depomed, Inc. |
| 2010/11/2 | Bevacizumab | D06409 | Treatment of primary peritoneal carcinoma. | AVASTIN | Either in combination with carboplatin and paclitaxel or in combination with carboplatin and gemcitabine, followed by Avastin as a single agent, is indicated for the treatment of patients with platinum-sensitive recurrent epithelial ovarian, fallopian tube, or primary peritoneal cancer. | 2016/12/6 | Genentech, Inc. |
| In combination with paclitaxel, pegylated liposomal doxorubicin, or topotecan for treatment of patients with platinum-resistant, recurrent epithelial ovarian, fallopian tube, or primary peritoneal cancer who received no more than 2 prior chemotherapy regimens | 2014/11/14 | ||||||
| 2010/9/13 | Crizotinib | D09731 | Treatment of ALK-positive, MET-positive, or ROS-positive non-small cell lung cancer | XALKORI | Treatment of patients with locally advanced or metastatic non-small cell lung cancer (NSCLC) that is anaplastic lymphoma kinase (ALK)-positive as detected by an FDAapproved test | 2011/8/26 | Pfizer, Inc. |
| 2010/9/2 | Dichlorphenamide | D00518 | Treatment of primary periodic paralyses | KEVEYIS | Treatment of primary hyperkalemic periodic paralysis, primary hypokalemic period paralysis, and related variants | 2015/8/7 | Taro Pharmaceuticals |
| 2010/8/19 | Anakinra | D02934 | Treatment of cryopyrin-associated periodic syndromes | KINERET | Treatment of neonatal-onset multisystem inflammatory disease (NOMID) | 2012/12/21 | Swedish Orphan Biovitrum AB (publ) |
| 2010/6/17 | Treprostinil (inhalational) | D06213 | Treatment of pulmonary arterial hypertension | TYVASO | Treatment of pulmonary arterial hypertension (WHO Group I) in patients with NYHA Class III symptoms, to increase walk distance | 2009/7/30 | LungRx, Inc. |
| 2010/4/30 | Cinacalcet | D03505 | Treatment of hypercalcemia in patients with primary hyperparathyroidism for whom parathyroidectomy would be indicated on the basis of serum calcium levels, but who are unable to undergo surgery | SENSIPAR | Treatment of severe hypercalcemia in patients with primary hyperparathyroidism who are unable to undergo parathyroidectomy | 2011/2/25 | Amgen, Inc. |
| 2010/4/30 | Cinacalcet | D03505 | Treatment of hypercalcemia in patients with primary hyperparathyroidism for whom parathyroidectomy would be indicated on the basis of serum calcium levels, but who are unable to undergo surgery | SENSIPAR | Treatment of hypercalcemia in adult patients with primary hyperparathyroidism for whom parathyroidectomy would be indicated on the basis of serum calcium levels, but who are unable to undergo parathyroidectomy. | 2014/11/21 | Amgen, Inc. |
| 2010/4/30 | Selexipag | D09994 | Treatment of pulmonary arterial hypertension | UPTRAVI | For use of Uptravi (Selexipag) Tablets, 200, 400, 600, 800, 1000, 1200, 1400, and 1600 mcg for treatment of pulmonary arterial hypertension (PAH, WHO Group I) to reduce the risks of disease progression and hospitalization for PAH | 2015/12/21 | Actelion Ltd |
| 2010/3/26 | Ruxolitinib | D09960 | Treatment of polycythemia vera | JAKAFI | Treatment of patients with polycythemia vera who have had an inadequate response to or are intolerant of hydroxyurea. | 2014/12/4 | Incyte Corporation |
| 2010/1/19 | Tasimelteon | D09388 | Non-24-hour sleepwake disorder in blind individuals without light perception | HETLIOZ | Treatment of non-24-hour sleep-wake disorder | 2014/1/31 2015/7/3 (EU) |
Vanda Pharmaceuticals, Inc. Vanda Pharmaceuticals Ltd (EU) |
| 2009/11/20 | Ponatinib | D09951 | Treatment of chronic myeloid leukemia | ICLUSIG | Treatment of adult patients with chronic phase, accelerated phase, or blast phase chronic myeloid leukemia (CML) that is resistant or intolerant to prior tyrosine kinase inhibitor therapy. | 2012/12/14 | ARIAD Pharmaceuticals Inc. |
| 2009/11/20 | Ponatinib | D09951 | Treatment of Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ALL) | ICLUSIG | Treatment of adult patients with Philadelphia chromosome positive acute lymphoblastic leukemia (Ph+ALL) that is resistant or intolerant to prior tyrosine kinase inhibitor therapy. | 2012/12/14 | ARIAD Pharmaceuticals Inc. |
| 2009/10/20 | Pazopanib | D05380 | Treatment of soft tissue sarcomas | VOTRIENT | Advanced soft tissue sarcoma (STS) who have received prior chemotherapy | 2012/4/26 | Novartis Pharmaceuticals Corp. |
| 2009/10/13 | Trastuzumab | D03257 | Treatment of HER2-overexpressing advanced adenocarcinoma of the stomach, including gastroesophageal junction | HERCEPTIN | Treatment of patients with HER2 overexpressing metastatic gastric or gastroesophageal junction adenocarcinoma, who have not received prior treatment for metastatic disease | 2010/10/20 | Genentech, Inc. |
| 2009/9/3 | Belinostat | D08870 | Treatment of peripheral T-cell lymphoma (PTCL). | BELEODAQ | Treatment of patients with relapsed or refractory peripheral T-cell lymphoma | 2014/7/3 | Spectrum Pharmaceuticals, Inc. |
| 2009/9/3 | Macitentan | D10135 | Treatment of pulmonary arterial hypertension | OPSUMIT | Treatment of pulmonary arterial hypertension (PAH, WHO Group 1) to delay disease progression. Disease progression included: death, initiation of intravenous (IV) or subcutaneous prostanoids, or clinical worsening of PAH (decreased 6-minute walk distance, worsened PAH symptoms and need for additional PAH treatment). Opsumit also reduced hospitalization for PAH. | 2013/10/18 2013/12/20 (EU) |
Actelion Pharmaceuticals Ltd Actelion Registration Ltd (EU) |
| 2009/9/3 | Paclitaxel protein-bound particles | D00491 | Treatment of pancreatic cancer. | ABRAXANE | Treatment of metastatic adenocarcinoma of the pancreas as first-line treatment, in combination with gemcitabine. | 2013/9/6 | Abraxis BioScience, LLC |
| 2009/9/3 | Taliglucerase alfa | D09675 | Treatment of Gaucher's disease | ELELYSO | Use as long-term enzyme replacement therapy in patients with Type 1 Gaucher disease | 2012/5/1 | Pfizer, Inc. |
| 2009/8/25 | Pasireotide | D10566 | Treatment of acromegaly | SIGNIFOR LAR | Treatment of patients with acromegaly who have had an inadequate response to surgery and/or for whom surgery is not an option | 2014/12/15 2012/4/24 (EU) |
Novartis Pharmaceuticals Corporation Novartis Europharm Limited (EU) |
| 2009/7/24 | Pasireotide | D10497 | Treatment of Cushing's disease | SIGNIFOR | Treatment of adult patients with Cushing's disease for whom pituitary surgery is not an option or has not been curative | 2012/12/14 | Novartis Pharmaceuticals Corporation |
| 2009/6/8 | Everolimus | D02714 | Treatment of tuberous sclerosis complex including TSC-associated subependymal giant cell astrocytoma (SEGA), TSC-associated angiomyolipoma and TSC-associated lymphangioleiomyomatosis (LAM) | AFINITOR VOTUBIA (EU) |
Treatment of adults with renal angiomyolipoma and tuberous sclerosis complex (TSC) not requiring immediate surgery. | 2012/4/26 2011/9/2 (EU) |
Novartis Pharmaceuticals Corporation Novartis Europharm Ltd (EU) |
| 2009/6/8 | Everolimus | D02714 | Treatment of tuberous sclerosis complex including TSC-associated subependymal giant cell astrocytoma (SEGA), TSC-associated angiomyolipoma and TSC-associated lymphangioleiomyomatosis (LAM) | AFINITOR | Treatment of patients with subependymal giant cell astrocytoma (SEGA) associated with tuberous sclerosis who require therapeutic intervention but are not candidates for curative surgical resection | 2010/10/29 | Novartis Pharmaceuticals Corporation |
| 2009/6/8 | Velaglucerase-alfa | D09029 | Treatment of Gaucher disease | VPRIV | Treatment of Type I Gaucher Disease | 2010/2/26 2010/8/26 (EU) |
Shire Human Genetics Therapies, Inc. Shire Pharmaceuticals Ireland Ltd (EU) |
| 2009/5/22 | Capsaicin | D00250 | Management of neuropathic pain in patients with postherpetic neuralgia | QUTENZA | Management of neuropathic pain associated with postherpetic neuralgia | 2009/11/16 | Acorda Therapeutics, Inc. |
| 2009/5/15 | Elosulfase alfa | D10333 | Use in the treatment of mucopolysaccharidosis (MPS) Type IV A (Morquio A syndrome) | VIMIZIM | Patients with Mucopolysaccharidosis type IVA (MPS IVA; Morquio A syndrome) | 2014/2/14 2014/4/28 (EU) |
BioMarin Pharmaceutical Inc. BioMarin Europe Ltd (EU) |
| 2009/4/29 | Eculizumab | D03940 | Treatment of atypical hemolytic uremic syndrome | SOLIRIS | For the treatment of atypical Hemolytic Uremic Syndrome (aHUS) | 2011/9/23 | Alexion Pharmaceuticals, Inc. |
| 2009/4/27 | Glycerol phenylbutyrate | D10127 | Maintenance treatment of patients with deficiencies in enzymes of the urea cycle | RAVICTI | Use as a nitrogen-binding adjunctive therapy for chronic management of adult and pediatric patients at least 2 years of age with urea cycle disorders (UCDs) that cannot be managed by dietary protein restriction and/or amino acid supplementation alone. RAVICTI must be used with dietary protein restriction and, in some cases, dietary supplements (eg, essential amino acids, arginine, citrulline, protein-free calorie supplements). | 2013/2/1 2015/11/27 (EU) |
Hyperion Therapeutics, Inc. Horizon Therapeutics Limited (EU) |
| 2009/4/27 | Lenalidomide | D04687 | Treatment of mantle cell lymphoma | REVLIMID | Treatment of mantle cell lymphoma whose disease has relapsed or progressed after two prior therapies, one of which included bortezomib. | 2013/6/5 | Celgene Corporation |
| 2009/3/17 | Technetium Tc99m sulfur colloid injection, lyophilized | D06048 | For localization of sentinel lymph nodes in patients with melanoma. | TECHNETIUM TC99M SULFUR COLLOID | Localization of lymph nodes draining a primary tumor in patients with melanoma when used with a hand-held gamma counter | 2012/8/13 | Pharmalucence, Inc. |
| 2009/3/17 | Tenofovir | D01982 | Treatment of pediatric HIV infection. | VIREAD | Treatment of HIV infection in combination with other antiretroviral agents in patients 12 to less than 18 years of age | 2010/3/24 | Gilead Sciences, Inc. |
| 2009/3/10 | Ofatumumab | D09314 | Treatment of chronic lymphocytic leukemia | ARZERRA | Arzerra in combination with fludarabine and cyclophosphamide for the treatment of patients with relapsed chronic lymphocytic leukemia (CLL). | 2016/8/30 | Novartis Pharmaceuticals Corp. |
| For extended treatment of patients who are in complete or partial response after at least two lines of therapy for recurrent or progressive CLL. | 2016/1/19 | Novartis Pharmaceuticals Corp. | |||||
| Ofatumumab in combination with chlorambucil, for the treatment of previously untreated patients with chronic lymphocytic leukemia (CLL) for whom fludarabine-based therapy is considered inappropriate. | 2014/4/17 | Novartis Pharmaceuticals Corp. | |||||
| Treatment of chronic lymphocytic leukemia (CLL) refractory to alemtuzumab and fludarabine | 2009/10/26 2010/4/19 (EU) |
Novartis Pharmaceuticals Corp. Novartis Europharm Ltd (EU) |
|||||
| 2009/2/24 | Bosutinib | D09728 | Treatment of chronic myelogenous leukemia | BOSULIF | Treatment of adult patients with chronic, accelerated or blast phase Philadelphia chromosome-positive (Ph+) chronic myelogenous leukemia (CML) with resistance, or intolerance to prior therapy | 2012/9/4 2013/3/27 (EU) |
Wyeth Pharmaceuticals, Inc. Pfizer Ltd (EU) |
| 2008/11/24 | Melphalan hydrochloride | D08173 | High dose conditioning treatment prior to hematopoietic progenitor (stem) cell transplantatio | EVOMELA | for high-dose conditioning treatment prior to hematopoietic progenitor (stem) cell transplantation in patients with multiple myeloma | 2016/3/10 | Spectrum Pharmaceuticals, Inc. |
| 2008/10/30 | Coagulation factor IX (recombinant), Fc fusion protein | Control and prevention of hemorrhagic episodes in patients with hemophilia B (congenital factor IX deficiency or Christmas disease) | ALPROLIX | Adults and children with Hemophilia B for control and prevention of bleeding episodes, perioperative management, and routine prophylaxis to prevent or reduce the frequency of bleeding episodes. | 2014/3/28 | Biogen Idec Inc. | |
| 2008/10/23 | Brentuximab vedotin | D09587 | Treatment of anaplastic large cell lymphoma | ADCETRIS | The treatment of patients with systemic anaplastic large cell lymphoma (sALCL) after failure of at least one prior multi-agent chemotherapy regimen | 2011/8/19 2012/10/25 (EU) |
Seattle Genetics, Inc. Takeda Pharma A/S (EU) |
| 2008/9/30 | Canakinumab | D09315 | Treatment of pediatric (age 16 and under) juvenile rheumatoid arthritis. | ILARIS | Treatment of active Systemic Juvenile Idiopathic Arthritis (SJIA) in patients aged 2 through 16 years. | 2013/5/9 | Novartis Pharmaceuticals Corporation |
| 2008/9/30 | Difluprednate | D01266 | Treatment of endogenous and traumatic anterior uveitis and panuveitis. | DUREZOL | Treatment of endogenous anterior uveitis | 2012/6/13 | Alcon Pharmaceuticals. Ltd. |
| 2008/9/17 | Eliglustat | D09893 | Treatment of Type I Gaucher disease | CERDELGA | Long-term treatment of adult patients with Gaucher disease type 1 who are CYP2D6 extensive metabolizers (EMs), intermediate metabolizers (IMs), or poor metabolizers (PMs) as detected by an FDA-cleared test. | 2014/8/19 2015/1/19 (EU) |
Genzyme Corporation Genzyme Europe BV (EU) |
| 2008/9/5 | Propranolol | D00483 | Treatment of proliferating infantile hemangiomas requiring systemic therapy | HEMANGEOL | Treatment of proliferating infantile hemangioma requiring systemic therapy. | 2014/3/14 | Pierre Fabre Dermatologie |
| 2008/9/5 | Ruxolitinib phosphate | D09960 | Treatment of myelofibrosis | JAKAFI | Treatment of patients with intermediate or high-risk myelofibrosis, including primary myelofibrosis, post-polycythemia vera myelofibrosis and post-essential thrombocythemia myelofibrosis | 2011/11/16 | Incyte Corporation |
| 2008/7/29 | Anthrax immune globulin (human) | Treatment of inhalational anthrax | Treatment of inhalational anthrax in adult and pediatric patients in combination with appropriate antibacterial drugs | 2015/3/24 | Cangene Corp. - Emergent Biosolutions | ||
| 2008/7/25/ | Sotalol (IV) | For ventricular tachycardia, ventricular fibrillation, or the maintenance of sinus rhythm in patients converted from atrial fibrillation or atrial flutter when oral administration is not possible. | SO-AQUEOUS | For use as a substitute for oral sotalol in patients who are unable to take sotalol orally [oral sotalol is indicated for maintenance of normal sinus rhythm in patients with history of highly symptomatic atrial fibrillation /flutter, and for treatment of documented life-threatening ventricular arrhythmias] | 2009/7/2 | Academic Pharmaceuticals | |
| 2008/5/16 | Blinatumomab | D09325 | Treatment of acute lymphocytic leukemia | BLINCYTO | Treatment of Philadelphia chromosome-negative relapsed or refractory B-cell precursor acute lymphoblastic leukemia (ALL). | 2014/12/3 2015/11/23 (EU) |
Amgen, Inc. Amgen Europe B.V. (EU) |
| 2008/5/5 | Eltrombopag | D03978 | Treatment of idiopathic thrombocytopenia purpura | PROMACTA | Treatment of thrombocytopenia in patients with chronic immune (idiopathic) thrombocytopenic purpura who have had an insufficient response to corticosteroids, immunoglobulins, or splenectomy | 2008/11/20 | Novartis Pharmaceuticals Corp. |
| 2008/4/9 | Obeticholic acid | D09360 | Treatment of primary biliary cirrhosis | OCALIVA | Treatment of primary biliary cholangitis (PBC) in combination with ursodeoxycholic acid (UDCA) in adults with an inadequate response to UDCA, or as monotherapy in adults unable to tolerate UDCA | 2016/5/27 | Intercept Pharmaceuticals, Inc. |
| 2008/4/9 | Peginterferon alfa-2b | D02748 | Treatment of malignant melanoma stages IIb through IV. | SYLATRON | Adjuvant treatment of melanoma with microscopic or gross nodal involvement within 84 days of definitive surgical resection including complete lymphadenectomy | 2011/3/29 | Schering-Plough Corporation |
| 2008/4/9 | Sodium nitrite and sodium thiosulfate | Treatment of known or suspected cyanide poisoning | NITHIODOTE | For sequential use for the treatment of cyanide poisoning that is judged to be life-threatening | 2011/1/14 | Hope Pharmaceuticals | |
| 2008/3/24 | Hepatitis B immune globulin (human) | D04431 | Prevention of hepatitis B recurrence following orthotopic liver transplant | HEPAGAM | Prevention of hepatitis B recurrence following liver transplantation in HBsAG-positive liver transplant patients | 2007/4/6 | Cangene Corporation |
| 2008/3/13 | Human fibrinogen concentrate, pasteurized | Treatment of fibrinogen deficient patients. | RIASTAP | Treatment of acute bleeding episodes in patients with congenital fibrinogen deficiency (afibrinogenemia and hypofibrinogenemia) | 2009/1/16 | CSL Behring, LLC | |
| 2008/2/20 | Belatacept | D03222 | Prophylaxis of organ rejection in renal allograft recipients | NULOJIX | Prophylaxis of organ rejection in adult patients receiving kidney transplants | 2011/6/15 | Bristol-Myers Squibb Company |
| 2008/2/14 | Everolimus | D02714 | Treatment of neuroendocrine tumors | AFINITOR | Treatment of progressive neuroendocrine tumors of pancreatic origin (PNET) in patients with unresectable, locally advanced or metastatic disease | 2011/5/5 | Novartis Pharmaceuticals Corporation |
| Treatment of adult patients with progressive, well-differentiated, non-functional, neuroendocrine tumors (NET) of gastrointestinal (GI) or lung origin, (excluding pancreatic) with unresectable, locally advanced or metastatic disease. | 2016/2/26 | ||||||
| 2008/1/18 | Carfilzomib | D08880 | Treatment of multiple myeloma | KYPROLIS | Treatment of patients with multiple myeloma who have received at least two prior therapies including bortezomib and an immunomodulatory agent and have demonstrated disease progression on or within 60 days of completion of the last therapy. | 2012/7/20 2015/11/19 |
Onyx Therapeutics, Inc. Amgen Europe B.V. (EU) |
| 2008/1/8 | Mitomycin-C | D00208 | Treatment of refractory glaucoma as an adjunct to ab externo glaucoma surgery. | MITOSOL | An adjunct to ab externo glaucoma surgery. | 2012/2/07 | Mobius Therapeutics, LLC |
| 2007/12/19 | Coccidioidin SD Skin Test Antigen | D03576 | For the diagnosis of Coccidioidomycosis | For the detection of delayed type hypersensitivity to C. immitis in individuals, 18-64 years of age, with a history of pulmonary coccidioidomy-cosis | 2011/7/29 | Allermed Labortories, Inc. | |
| 2007/12/18 | Fludarabine phosphate oral tablets | D01907 | Treatment of B-cell chronic lymphocytic leukemia | Treatment of adult patients with B-cell chronic lymphocytic leukemia (CLL) whose disease has not responded to or has progressed during or after treatment with at least one standard alkylating-agent containing regimen | 2008/12/18 | Sanofi-Aventis U.S., Inc. | |
| 2007/12/18 | Canakinumab | D09315 | Treatment of cryopyrin-associated periodic syndromes | ILARIS | Treatment of cryopyrin-associated periodic syndromes (CAPS), in adults and children 4 years of age and older. | 2009/6/17 | Novartis Pharmaceuticals Corporation |
| 2007/12/18 | Clobazam | D01253 | Treatment of Lennox-Gastaut Syndrome | ONFI | Adjunctive treatment of seizures associated with Lennox-Gastaut Syndrome inpatients 2 years of age or older | 2011/10/21 | Lundbeck, Inc. |
| 2007/12/7 | Recombinant human antithrombin | D08858 | Treatment of congenital antithrombin deficiency to prevent the occurrence of serious, potentially life-threatening venous thromboembolisms which may develop as a result of surgical or obstetrical procedures | ATRYN | Prevention of peri-operative and peri-partum thromboembolic events, in hereditary antithrombin deficient patients. | 2009/2/6 | GTC Biotherapeutics, Inc. |
| 2007/10/23 | Eteplirsen | D09900 | Treatment of Duchenne Muscular Dystrophy | EXONDYS 51 | Treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 51 skipping | 2016/9/16 | Sarepta Therapeutics, Inc. |
| 2007/10/23 | Lomitapide | D09638 | Treatment of homozygous familial hypercholesterolemia | JUXTAPID | Adjunct to a low-fat diet and other lipid-lowering treatments, including LDL apheresis where available, to reduce low-density lipoprotein cholesterol (LDL-C), total cholesterol (TC), apolipoprotein B (apo B), and non-high-density lipoprotein cholesterol (non-HDL-C) in patients with homozygous familial hypercholesterolemia (HoFH). | 2012/12/21 | Aegerion Pharmaceuticals, Inc. |
| 2007/9/25 | Colchicine | D00570 | Treatment of familial Mediterranean fever | COLCRYS | Treatment of familial Mediterranean fever | 2009/7/29 | AR Holding Company, Inc. |
| 2007/8/31 | Artemether/lumefantrine | D02484 | For the treatment of infections due to Plasmodium falciparum or mixed infections including P. falciparum. | COARTEM | Treatment of malaria in patients of 5 kg bodyweight or above with acute, uncomplicated malaria due to Plasmodium falciparum or mixed infections including P. falciparum | 2009/4/7 | Novartis Pharmaceuticals |
| 2007/8/31 | Parathyroid hormone | D05364 | Treatment of hypoparathyroidism | NATPARA | An adjunct to calcium and vitamin D to control hypocalcemia in patients with hypoparathyroidism | 2015/1/23 | NPS Pharmaceuticals, Inc. |
| 2007/8/17 | Bendamustine hydrochloride | D07085 | Treatment of chronic lymphocytic leukemia | TREANDA | Treatment of patients with chronic lymphocytic leukemia | 2008/3/20 | Cephalon, Inc. |
| 2007/7/5 | Mifepristone | D00585 | Treatment of the clinical manifestations of endogenous Cushing's syndrone | KORLYM | for the control of hyperglycemia secondary to hypercortisolism in adult patients with endogenous Cushing's syndrome who have type 2 diabetes mellitus or glucose intolerance and have failed surgery or are not candidates for surgery. | 2012/2/17 | Corcept Therapeutics, Inc. |
| 2007/4/18 | Human plasma coagulation Factor VIII and human plasma von Willebrand Factor | Treatment of von Willebrand disease except for surgical and/or invasive procedures in patients with von Willebrand disease in whom desmopressin is either ineffective or contraindicated | WILATE | Treatment of spontaneous or trauma-induced bleeding episodes in patients with severe von Willebrand disease (VWD) as well as patients with mild or moderate VWD in whom the use of desmopressin is known or suspected to be ineffective or contraindicated | 2009/12/4 | Octapharma USA, Inc. | |
| 2007/3/22 | Ganciclovir | D00333 | Treatment of acute herpetic keratitis (dendritic and geographic ulcers) | ZIRGAN | Treatment of acute herpetic keratitis (dendritic ulcers) | 2009/9/15 | Sirion Therapeutics, Inc. |
| 2007/1/30 | Brentuximab vedotin | D09587 | Treatment of Hodgkin's lymphoma | ADCETRIS | The treatment of patients with Hodgkin lymphoma after failure of autologous stem cell transplant (ASCT) or after failure of at least two prior multi-agent chemotherapy regimens in patients who are not ASCT candidates | 2011/8/19 2012/10/25 (EU) |
Seattle Genetics, Inc. Takeda Pharma A/S (EU) |
| 2007/1/25 | Hydroxyprogesterone caproate | D00949 | Prevention of preterm birth in singleton pregnancies | MAKENA | To reduce the risk of preterm birth in women with singleton pregnancy who have a history of singleton spontaneous preterm birth | 2011/2/3 | KV Pharmaceutical Company |
| 2007/1/17 | Droxidopa | D01277 | Treatment of neurogenic symptomatic orthostatic hypotension in patients with primary autonomic failure, dopamine-beta-hydroxylase deficiency, and nondiabetic autonomic neuropathy. | NORTHERA | Treatment of orthostatic dizziness, lightheadedness, or the "feeling that you are about to black out" in adult patients with symptomatic neurogenic orthostatic hypotension caused by primary autonomic failure (Parkinson's disease, multiple system atrophy, and pure autonomic failure), dopamine beta-hydroxylase deficiency, and non-diabetic autonomic neuropathy. | 2014/2/18 | Lundbeck LLC |
| 2007/1/8 | Vincristine sulfate LIPOSOME injection | D02197 | Treatment of acute lymphoblastic leukemia | MARQIBO | Treatment of adult patients with Philadelphia chromosome-negative (Ph-) acute lymphoblastic leukemia (ALL) in second or greater relapse or whose disease has progressed following two or more anti-leukemia therapies. | 2012/8/9 | Talon Therapeutics, Inc. |
| 2006/12/20 | Ivacaftor | D09916 | Treatment of patients with cystic fibrosis | KALYDECO | Treatment of cystic fibrosis (CF) in patients age 6 years and older who have a G551D mutation in the CFTR gene. | 2012/1/31 2012/7/23 (EU) |
Vertex Pharmaceuticals, Inc. Vertex Pharmaceuticals (Europe) Ltd (EU) |
| 2006/12/18 | Tadalafil | D02008 | Treatment of pulmonary arterial hypertension | ADCIRCA | Treatment of pulmonary arterial hypertension (WHO Group I) to improve exercise ability | 2009/5/22 | Eli Lilly and Company |
| 2006/12/1 | Iobenguane I 123 | D04559 | For the diagnosis of pheochromocytomas | ADREVIEW | To be used in the detection of primary or metastatic pheochromocytomas or neuroblastomas as an adjunct to other diagnostic tests | 2008/9/19 | GE Healthcare, Inc. |
| 2006/12/1 | Iobenguane I 123 | D04559 | For the diagnosis of neuroblastomas | ADREVIEW | To be used in the detection of primary or metastatic pheochromocytomas or neuroblastomas as an adjunct to other diagnostic tests | 2008/9/19 | GE Healthcare, Inc. |
| 2006/11/7 | Varicella Zoster Immune Globulin (Human) | D06284 | Passive immunization for the treatment of exposed, susceptible individuals who are at risk of complications from varicella | VARIZIG | Post exposure prophylaxis of varicella in high risk individuals to reduce the severity of varicella | 2012/12/20 | Cangene bioPharma, Inc. |
| 2006/10/24 | Cysteamine enteric coated | D10468 | Treatment of cystinosis | PROCYSBI | To expand the indication to pediatric patients 2-6 years of age with nephropathic cystinosis | 2015/8/14 | Raptor Therapeutics, Inc. |
| 2006/10/24 | Cysteamine enteric coated | D10468 | Treatment of cystinosis | PROCYSBI | For management of nephropathic cystinosis in adults and children ages 6 years and older | 2013/4/30 | Raptor Therapeutics, Inc. |
| 2006/10/19 | Adalimumab | D02597 | Treatment of pediatric Crohn's disease. | HUMIRA | Reducing signs and symptoms and inducing and maintaining clinical remission in patients 6 years of age and older with moderately to severely active Crohn's disease who have had an inadequate response to corticosteroids or immunomodulators such as azathioprine, 6-mercaptopurine, or methotrexate. (Orphan approval for patients from 6 through 16 years of age.) | 2014/9/23 | AbbVie, Inc. |
| 2006/10/10 | Miltefosine | D02494 | Treatment of leishmaniasis. | IMPAVIDO | Treatment of visceral leishmaniasis due to Leishmania donovani; cutaneous leishmaniasis due to Leishmania braziliensis, Leishmania guyanensis, and Leishmania panamensis; and mucosal leishmaniasis due to Leishmania braziliensis. | 2014/3/19 | Knight Therapeutics (USA) |
| 2006/8/9 | Somatropin | D02691 | Treatment of short stature in patients with Noonan syndrome | NORDITROPIN | Treatment of short stature in patients with Noonan syndrome | 2007/5/31 | Novo Nordisk Inc. |
| 2006/8/2 | Trypan blue | C19307 | Selectively staining epiretinal membranes during ophthalmic surgical vitrectomy procedures | MEMBRANEBLUE | For use as an aid in ophthalmic surgery by staining the epiretinal membranes during ophthalmic surgical vitrectomy procedures, facilitating removal of the tissue | 2009/2/20 | Dutch Ophthalmic Research Center Int'l BV |
| 2006/7/20 | Immune globulin infusion (human) | D06458 | Treatment of multifocal motor neuropathy | GAMMAGARD | Maintenance therapy to improve muscle strength and disability in adult patients with Multifocal Motor Neuropathy (MMN). | 2012/6/22 | Baxter Healthcare Corporation |
| 2006/7/20 | Pralatrexate | D05589 | Treatment of T-cell lymphoma | FOLOTYN | Treatment of patients with relapsed or refractory peripheral T-cell lymphoma | 2009/9/25 | Allos Therapeutics, Inc. |
| 2006/6/9 | Glycopyrrolate | D00540 | Treatment of pathologic (chronic moderate to severe) drooling in pediatric patients | CUVPOSA | To reduce chronic drooling in patients aged 3 - 16 with neurologic conditions associated with problem drooling (e.g. cerebral palsy) | 2010/7/28 | Shionogi, Inc. |
| 2006/6/9 | Obiltoxaximab | D10776 | Treatment of exposure to B. anthracis spores | ANTHIM | Treatment of adult and pediatric patients with inhalational anthrax due to Bacillus anthracis in combination with appropriate antibacterial drugs and for prophylaxis of inhalational anthrax when alternative therapies are not available or are not appropriate | 2016/3/18 | Elusys Therapeutics, Inc. |
| 2006/5/26 | Bevacizumab | D06409 | Treatment of malignant glioma | AVASTIN | Treatment of glioblastoma with progressive disease following prior therapy | 2009/5/5 | Genentech, Inc. |
| 2006/5/26 | Siltuximab | D09669 | Treatment of Castleman's disease | SYLVANT | Treatment of patients with multicentric Castleman's disease (MCD) who are human immunodeficiency virus (HIV) negative and human herpesvirus-8 (HHV-8) negative. | 2014/4/23 2014/5/22 (EU) |
Janssen Biotech, Inc. Janssen-Cilag International NV (EU) |
| 2006/5/23 | Mipomersen | D08946 | Treatment of homozygous familial hypercholesterolemia | KYNAMRO | Adjunct to lipid-lowering medications and diet to reduce low density lipoprotein-cholesterol (LDL-C), apolipoprotein B (apo B), total cholesterol (TC), and non-high density lipoprotein-cholesterol (non-HDL-C) in patients with homozygous familial hypercholesterolemia (HoFH). | 2013/1/29 | Genzyme Corporation |
| 2006/4/27 | Nilotinib | D06413 | Treatment of chronic myelogenous leukemia | TASIGNA | For the use for chronic phase (CP) and accelerated phase (AP) Philadelphia chromosome positive chronic myelogenous leukemia (CML) in adult patients resistant to or intolerant to prior therapy that included imatinib. | 2007/10/29 2007/11/19 (EU) |
Novartis Pharmaceutical Corporation Novartis Europharm Ltd (EU) |
| 2006/4/20 | Sorafenib | D06272 | Treatment of hepatocellular carcinoma | NEXAVAR | Treatment of unresectable hepatocellular carcinoma | 2007/11/16 2006/7/19 (EU) |
Bayer Pharmaceuticals Corporation Bayer Pharma AG (EU) |
| 2006/3/10 | Omacetaxine mepesuccinate | D08956 | Treatment of chronic myelogenous leukemia | SYNRIBO | Treatment of adult patients with chronic or accelerated phase chronic myeloid leukemia (CML) with resistance and/or intolerance to two or more tyrosine kinase inhibitors (TKI) | 2012/10/26 | IVAX International GmbH |
| 2006/2/14 | Rituximab | D02994 | Treatment of patients with anti-neutrophil cytoplasmic antibody-associated vasculitis (Wegener's Granulomatosis, Microscopic Polyangiitis, and Churg-Strauss Syndrome) | RITUXAN | For the use of Rituxan (rituximab) in combination with glucocorticoids for the treatment of patients with Wegener's Granulomatosis (WG) and Microscopic Polyangiitis (MPA). ) | 2011/4/19 | Genentech, Inc. |
| 2006/2/9 | Bevacizumab | D06409 | Therapeutic treatment of patients with ovarian cancer | AVASTIN | Either in combination with carboplatin and paclitaxel or in combination with carboplatin and gemcitabine, followed by Avastin as a single agent, is indicated for the treatment of patients with platinum-sensitive recurrent epithelial ovarian, fallopian tube, or primary peritoneal cance | 2016/12/6 | Genentech, Inc. |
| In combination with paclitaxel, pegylated liposomal doxorubicin, or topotecan for treatment of patients with platinum-resistant, recurrent epithelial ovarian, fallopian tube, or primary peritoneal cancer who received no more than 2 prior chemotherapy regimens | 2014/11/14 | ||||||
| 2006/1/25 | Aripiprazole | D01164 | Treatment of Tourette's syndrome | ABILIFY | Treatment of pediatric patients with Tourette's | 2014/12/12 | Otsuka Pharmaceutical Development |
| 2005/12/19 | Imatinib mesylate | D01441 | Treatment of dermatofibrosarcoma protuberans | GLEEVEC | Treatment of adult patients with unresectable, recurrent and/or metastatic dermatofibrosarcoma protuberans (DFSP) | 2006/10/19 | Novartis Pharmaceuticals Corporation |
| 2005/12/15 | Somatropin | D02691 | Treatment of short stature in pediatric patients with short stature homeobox-containing gene (SHOX) deficiency | HUMATROPE | Treatment of short stature or growth failure in children with SHOX (short stature homeobox-containing gene) deficiency whose epiphyses are not closed | 2006/11/1 | Eli Lilly and Company |
| 2005/1/28 | Dasatinib | D06414 | Treatment of chronic myelogenous leukemia | SPRYCEL | Treatment of adults with chronic myeloid leukemia with resistance or intolerance to prior therapy including imatinib | 2006/6/28 2006/11/20 (EU) |
Bristol-Myers Squibb Company Bristol-Myers Squibb Pharma EEIG (EU) |
| 2005/11/18 | Dasatinib | D06414 | Treatment of Philadelphia-positive acute lymphoblastic leukemia | SPRYCEL | Treatment of adults with Philadelphia chromosome-positive acute lymphoblastic leukemia with resistance or intolerance to prior therapy | 2006/6/28 2006/11/20 (EU) |
Bristol-Myers Squibb Company Bristol-Myers Squibb Pharma EEIG (EU) |
| 2005/11/18 | Histrelin | D02369 | Treatment of central precocious puberty | SUPPRELIN LA | Treatment of central precocious puberty | 2007/5/3 | Endo Pharmaceuticals Solutions, Inc. |
| 2005/10/21 | Vandetanib | D06407 | Treatment of patients with follicular thyroid carcinoma, medullary thyroid carcinoma, anaplastic thyroid carcinoma, and locally advanced and metastatic papillary thyroid carcinoma | CAPRELSA | Treatment of asymptomatic or progressive medullary thyroid cancer in patients with unresectable locally advanced or metastatic disease | 2011/4/6 | AstraZeneca Pharmaceutical LP |
| 2005/10/11 | Imatinib | D01441 | Treatment of Philadelphia-positive acute lymphoblastic leukemia | GLEEVEC | Treatment of pediatric patients with newly diagnosed Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ ALL) in combination with chemotherapy, approved under NDA #21588/S-037 | 2013/1/25 | Novartis Pharmaceuticals Corporation |
| 2005/10/11 | Imatinib | D01441 | Treatment of Philadelphia-positive acute lymphoblastic leukemia | GLEEVEC | Treatment of adult patients with relapsed or refractory Philadelphia chromosome positive acute lymphoblastic leukemia (Ph+ALL) | 2006/10/19 | Novartis Pharmaceuticals Corporation |
| 2005/10/5 | Imatinib mesylate | D01441 | Treatment of myeloproliferative disorders/myelodysplastic syndromes associated with platelet-derived growth factor gene re-arrangements | GLEEVEC | Treatment of adult patients with myelodysplastic/myeloproliferative diseases (MDS/MPD) associated with PDGFR (platelet-derived growth factor receptor) gene re-arrangements | 2006/10/19 | Novartis Pharmaceuticals Corporation |
| 2005/9/9 | Imatinib mesylate | D01441 | Treatment of systemic mastocytosis without the D816V c-kit mutation | GLEEVEC | Treatment of adult patients with aggressive mastocytosis (ASM) without the D816V c-Kit mutation or with c-Kit mutational status unknown | 2006/10/19 | Novartis Pharmaceuticals Corporation |
| 2005/8/25 | Imatinib mesylate | D01441 | Treatment of idiopathic hypereosinophilic syndrome including acute and chronic eosinophilic leukemia | GLEEVEC | Treatment of adult patients with hypereosinophic syndrome (HES) and/or chronic eosinophilic leukemia (CEL) who have the FIP1L1-PDGFRA ± fusion kinase (mutational analysis or FISH demonstration of CHIC2 allele deletion) and for patients with HES and/or CEL who are FIP1L1-PDGFRA ± fusion kinase negative or unknown | 2006/10/19 | Novartis Pharmaceuticals Corporation |
| 2005/8/12 | Balsalazide disodium | D02715 | Treatment of pediatric patients with ulcerative colitis | COLAZAL | Treatment of mildly to moderately active ulcerative colitis in patients 5 years of age and older. Safety and Effectiveness of Colazal beyond 8 weeks in children (ages 5-17) and 12 weeks in adults have not been established. | 2006/12/20 | Salix Pharmaceuticals, Inc. |
| 2005/8/9 | Bicarbonate infusate | C00288 | Use in the management of patients undergoing continuous renal replacement therapy with hemofiltration | NORMOCARB HF | Use as a replacement solution in Continuous Renal Replacement Therapy (CRRT) to replace water and to correct electrolytes and acid-base imbalances in adults and children | 2006/7/26 | Dialysis Solutions, Inc. |
| 2005/7/14 | Raloxifene | D02217 | Reduction of the risk of breast cancer in postmenopausal women | EVISTA | Reduction in risk of invasive breast cancer in postmenopausal women with osteoporosis and reduction in risk of invasive breast cancer in postmenopausal women at high risk for invasive breast cancer | 2007/9/13 | Eli Lilly and Company |
| 2005/6/6 | Tacrolimus | D00107 | Prophylaxis of organ rejection in patients receiving heart transplants. | PROGRAF | Prophylaxis of organ rejection in patients receiving allogenic heart transplants. | 2006/3/29 | Astellas Pharma US, Inc. |
| 2005/3/21 | Adalimumab | D02597 | Treatment of juvenile rheumatoid arthritis | HUMIRA | Reducing signs and symptoms of moderately to severely active polyarticular juvenile idiopathic arthritis in patients 2 years to 4 years of age. | 2014/9/30 | AbbVie Inc. |
| 2005/3/21 | Adalimumab | D02597 | Treatment of juvenile rheumatoid arthritis | HUMIRA | Reducing signs and symptoms of moderately to severely active polyarticular juvenile idiopathic arthritis in patients 4 years of age and older. | 2008/2/21 | AbbVie Inc. |
| 2005/1/10 | Bedaquiline; (1R,2S) 6-bromo-alpha-[2-(dimethylamino)ethyl]-2-methoxy-alpha-(1-naphthyl)-beta-phenyl-3-quinolineethanol | D09873 | Treatment of active tuberculosis | SIRTURO | Part of combination therapy in adults (greater than or equal to 18 years) with pulmonary multi-drug resistant tuberculosis (MDR-TB). | 2012/12/28 2014/3/5 (EU) |
Janssen Research & Development, LLC Janssen-Cilag International N.V. (EU) |
| 2004/12/29 | Doxorubicin HCL liposome injection | D01275 | Treatment of multiple myeloma | DOXIL | For use in combination with bortezomib for the treatment of patients with multiple myeloma who have not previously received bortezomib and have at least one prior therapy. | 2007/5/17 | Johnson & Johnson Pharmaceutical Research & Dev. |
| 2004/12/20 | Rilonacept | D06635 | Treatment of CIAS1-Associated Periodic Syndromes | ARCALYST | Treatment of Cryopyrin-Assisted Periodic Syndromes (CAPS) | 2008/2/27 | Regeneron Pharmaceuticals, Inc. |
| 2004/12/16 | Temsirolimus | D06068 | Treatment of renal cell carcinoma | TORISEL | Treatment of advanced renal cell carcinoma | 2007/5/30 2007/11/19 (EU) |
Wyeth Pharmaceuticals, Inc. Pfizer Limited (EU) |
| 2004/11/17 | Potassium Iodide Oral Solution | D01016 | For use as a thyroid blocking agent in pediatric patients exposed to radiactive iodine | THYROSHIELD | For use as a thyroid blocking agent in pediatric patients exposed to radiactive iodine | 2005/1/12 | Fleming & Company, Pharmaceuticals |
| 2004/10/8 | Rufinamide | D05775 | Treatment of Lennox-Gastaut Syndrome. | BANZEL INOVELON (EU) |
Adjunctive therapy of seizures associated with Lennox-Gastaut syndrome. | 2008/11/14 2007/1/16 (EU) |
Eisai, Inc. Eisai Ltd (EU) |
| 2004/10/8 | Sorafenib | D06272 | Treatment of renal cell carcinoma. | NEXAVAR | Treatment of patients with advanced renal cell carcinoma | 2005/12/20 2006/7/19 (EU) |
Bayer Pharmaceutical Corporation Bayer Pharma AG (EU) |
| 2004/9/30 | Romidepsin | D06637 | Treatment of non-Hodgkin T-cell lymphomas | ISTODAX | Treatment of peripheral T-cell lymphoma (PTCL) in patients who have received at least one prior therapy | 2011/6/16 | Celgene Corporation |
| 2004/9/30 | Romidepsin | D06637 | Treatment of non-Hodgkin T-cell lymphomas | ISTODAX | Treatment of cutaneous T-cell lymphoma (CTCL) in patients who have received at least one prior systemic therapy | 2009/11/5 | Celgene Corporation |
| 2004/9/10 | Coagulation factor VIIa (recombinant) | D00172 | Prevention of bleeding episodes in patients with congenital Factor VII deficiency | NOVOSEVEN | Prevention of bleeding in surgical interventions or invasive procedures in patients with congenital F VII deficiency | 2005/7/11 | Novo Nordisk, Inc. |
| 2004/9/10 | Coagulation factor VIIa (recombinant) | D00172 | Treatment of bleeding episodes in patients with congenital factor VII deficiency | NOVOSEVEN | Treatment of bleeding episodes in patients with Factor VII Deficiency | 2005/7/11 | Novo Nordisk, Inc. |
| 2004/8/17 | Mechlorethamine | D04872 | Treatment of mycosis fungoides | VALCHLOR | Topical treatment of Stage 1A and 1B mycosis fungoides-type cutaneous T-cell lymphoma in patients who have received prior skin-directed therapy. | 2013/8/23 | Actelion Pharmaceuticals Ltd. |
| 2004/8/17 | Iloprost inhalation solution | D02721 | Treatment of pulmonary arterial hypertension | VENTAVIS | Treatment of pulmonary arterial hypertension (WHO Group I) in patients with NYHA Class III or IV symptoms | 2004/12/29 | CoTherix, Inc. |
| 2004/8/10 | Nelarabine | D05134 | Treatment of acute lymphoblastic leukemia and lymphoblastic lymphoma | ARRANON ATRIANCE (EU) |
Treatment of patients with T-cell acute lymphoblastic leukemia and T-cell lymphoblastic lymphoma whose disease has not responded to or has relapsed following treatment with at least two chemotherapy regimens | 2005/10/28 2007/8/22 (EU) |
Novartis Pharmaceuticals Corp. Novartis Europharm Limited (EU) |
| 2004/7/27 | Immune Globulin (Human) | D06458 | Treatment of chronic inflammatory demyelinating polyneuropathy | GAMUNEX(R)-C | Treatment of Chronic Inflammatory Demyelinating Polyneuropathy (CIDP) to improve neuromuscular disability and impairment and for maintenance therapy to prevent relapse | 2008/9/12 | Grifols Therapeutics, Inc. |
| 2004/7/21 | Coagulation factor VIIa (recombinant) | D00172 | Prevention of bleeding episodes in patients with acquired inhibitors to Factor VIII or Factor IX | NOVOSEVEN | Prevention of bleeding in surgical interventions or invasive procedures in patients with acquired hemophilia | 2006/10/13 | Novo Nordisk, Inc. |
| 2004/7/16 | Coagulation Factor VIIa (Recombinant) | D00172 | Treatment of bleeding episodes in patients with acquired inhibitors to Factor VIII or Factor IX | NOVOSEVEN | Treatment of bleeding episodes in surgical interventions or invasive procedures in patients with acquired hemophilia | 2006/10/13 | Novo Nordisk, Inc. |
| 2004/7/16 | Ambrisentan | D07077 | Treatment of pulmonary arterial hypertension | LETAIRIS VOLIBRIS (EU) |
Treatment of pulmonary arterial hypertension (WHO group I) in patients with WHO class II or III symptoms to improve exercise capacity and delay clinical worsening | 2007/6/15 2008/4/21 (EU) |
Gilead Colorado Glaxo Group Ltd (EU) |
| 2004/7/16 | C1 esterase inhibitor (human) | Treatment of angioedema | CINRYZE(R) | Routine prophylaxis against angioedema attacks in patients with Hereditary Angioedema (HAE) | 2008/10/10 | Shire | |
| 2004/6/18 | Coagulation factor VIIa (recombinant) | D00172 | Treatment of bleeding episodes in Glanzmann's thrombasthenia | NOVOSEVEN RT | Treatment of bleeding episodes and peri-operative management in adults and children with Glanzmann's thrombasthenia with refractoriness to platelet transfusions, with or without antibodies to platelets. | 2014/7/2 | Novo Nordisk Inc. |
| 2004/6/18 | Coagulation factor VIIa (recombinant) | D00172 | Prevention of bleeding episodes in patients with hemophilia A or B, with or without inhibitors | NOVOSEVEN | Prevention of bleeding in surgical interventions or invasive procedures in hemophilia A or B patients with inhibitors to Factor VIII or Factor IX | 2005/8/12 | Novo Nordisk, Inc. |
| 2004/6/18 | Vaccinia Immune Globulin (Human) Intravenous | D06271 | Treatment of complications of vaccinia vaccination | CNJ-016 | treatment and/or modification of the following conditions, which are complications resulting from smallpox vaccination: Eczema vaccinatum Progressive vaccinia Severe generalized vaccinia Vaccinia infections in individuals who have skin conditions such as burns, impetigo, varicella-zoster, or poison ivy; or in individuals who have eczematous skin lesions because of either the activity or extensiveness of such lesions Aberrant infections induced by vaccinia virus that include its accidental implantation in eyes (except in cases of isolated keratitis), mouth, or other areas where vaccinia infection would constitute a special hazard. | 2005/5/2 | Cangene Corporation |
| 2004/6/18 | Vaccinia Immune Globulin (Human) Intravenous | D06271 | Treatment of severe complications from the smallpox vaccine | Treatment and modification of aberrant infections induced by vaccinia virus that include its accidental implantation in eyes (except in cases of isolated keratitis), mouth, or other areas where vaccinia infection would constitute a special hazard; eczema vaccinatum; progressive vaccinia; severe generalized vaccinia, and vaccinia infections in individuals who have skin conditions such as burns, impetigo, varicella-zoster, or poison ivy; or in individuals who have eczematous skin lesions because of either the activity or extensiveness of such lesions | 2005/2/18 | DynPort Vaccine Company LLC | |
| 2004/6/3 | Ipilimumab | D04603 | Treatment of high risk Stage II, Stage III, and Stage IV melanoma | YERVOY | Treatment of unresectable or metastatic melanoma | 2011/3/25 | Bristol-Myers Squibb Pharmaceutical Research Insti |
| 2004/6/3 | Quinine Sulfate | D02262 | Treatment of malaria | Treatment of uncomplicated Plasmodium falciparum malaria | 2005/8/12 | AR Holding Company, Inc. | |
| 2004/4/28 | Diethylenetriaminepentaacetic acid (DTPA) | Treatment of patients with known or suspected internal contamination with plutonium, americium, or curium to increase the rates of elimination. | Treatment of internal contamination with plutonium, americium or curium | 2004/8/11 | Hameln Pharmaceuticals gmbh | ||
| 2004/3/25 | Dexrazoxane | D07807 | Treatment of anthracycline extravasation during chemotherapy | TOTECT SAVENE (EU) |
Treatment of extravasation resulting from IV anthracycline chemotherapy | 2007/9/6 2006/7/28 (EU) |
Biocodex Clinigen Healthcare Ltd |
| 2004/3/16 | Antihemophilic factor (recombinant), porcine sequence | D02958 | Treatment and prevention of episodic bleeding in patients with inhibitor antibodies to human coagulation factor VIII | OBIZUR | Treatment of bleeding episodes in adults with acquired hemophilia A. | 10/23/2014 | Baxter Healthcare Corporation |
| 2004/3/16 | Vorinostat | D06320 | Treatment of T-cell non-Hodgkin's lymphoma | ZOLINZA | Treatment of cutaneous manifestations in patients with cutaneous T-cell lymphoma (CTCL) who have progressive, persistant or recurrent disease on or following two systemic therapies. | 2006/10/6 | Merck & Co., Inc. |
| 2004/3/8 | Multi-vitamin infusion without vitamin K | Prevention of vitamin deficiency and thromboembolic complications in people receiving home parenteral nutrition and warfarin-type anticoagulant therapy | M.V.I.-12 | Prevention of vitamin deficiency and thrombormbolic complications in people receiving home parenteral nutrition who also receive warfarin-type anticoagulant therapy | 2004/9/9 | Mayne Pharma (USA) Inc. | |
| 2004/3/5 | Pirfenidone | D01583 | Treatment of idiopathic pulmonary fibrosis | ESBRIET | Treatment of idiopathic pulmonary fibrosis | 2014/10/15 2011/2/28 (EU) |
Genentech Roche Registration Limited (EU) |
| 2004/1/29 | Antivenin crotaline (pit-viper) equine immune F(ab)2) | Treatment of envenomation by Crotaline snakes | ANTIVIPMYN | Management of adult and pediatric patients with North American rattlesnake envenomation | 2015/5/6 | Rare Disease Therapeutics, Inc. | |
| 2004/1/29 | Lenalidomide | D04687 | Treatment of myelodysplastic syndromes | REVLIMID | Treatment of patients with transfusion dependant anemia due to low or intermediate-1 risk myelodysplastic syndromes associated with a deletion 5 q cytogenetic abnormality with or without additional cytogenetic abnormalities | 2005/12/27 2007/6/14 (EU) |
Celgene Corporation Celgene Europe Ltd (EU) |
| 2004/1/29 | Rituximab | D02994 | Treatment of chronic lymphocytic leukemia | RITUXAN | Treatment of patients previously untreated for CD20-positive chronic lymphocytic leukemia in combination wih fludarbine and cyclophosphamide | 2010//2/18 | Genentech, Inc. |
| 2004/1/29 | Sapropterin | D01798 | Treatment of hyperphenylalaninemia | KUVAN | Indicated to reduce blood phenylalanine (Phe) levels in patients with hyperphenylalaninemia (HPA) due to tetrahydrobiopterin- (BH4-) responsive Phenylketonuria (PKU). Kuvan is to be used in conjunction with a Phe-restricted diet. | 2007/12/13 2008/12/2 (EU) |
BioMarin Pharmaceutical, Inc. Merck Serono Europe Limited (EU) |
| 2003/11/25 | Hydroxocobalamin | D01027 | Treatment of acute cyanide poisoning | CYANOKIT | Treatment on known or suspected cyanide poisoning | 2006/12/15 | SERB, S.A. |
| 2003/11/25 | Icatibant | D04492 | Treatment of angioedema | FIRAZYR | Treatment of acute attacks of hereditary angioedema in adults 18 years of age and older | 2011/8/25 2008/7/11 (EU) |
Shire Orphan Therapies Shire Orphan Therapies GmbH (EU) |
| 2003/11/12 | Infliximab | D02598 | Treatment of pediatric (0 to 16 years of age) ulcerative colitis | REMICADE | For reducing signs and symptoms and inducing and maintaining clinical remission in pediatric patients 6 years of age and older with moderately to severely active ulcerative colitis who have had an inadequate response to conventional therapy | 2011/9/23 | Janssen Biotech Inc. |
| 2003/11/12 | Infliximab | D02598 | Treatment of pediatric (0 to 16 years of age) Crohn's Disease | REMICADE | For reducing signs and symptoms and inducing and maintaining clinical remission in pediatric patients with moderately to severely active Crohn's disease who have had an inadequate response to conventional therapy | 2006/5/19 | Centocor, Inc. |
| 2003/11/12 | Raxibacumab | D05706 | Treatment of anthrax | ABTHARAXTM | Treatment of inhalation anthrax due to Bacillus anthracis in combination with appropriate antibacterial drugs, | 2012/12/14 | Human Genome Sciences, Inc. |
| 2003/11/6 | Bevacizumab | D06409 | Treatment of renal cell carcinoma | AVASTIN | Treatment of renal cell carcinoma in combination with interferon alfa | 2009/7/31 | Genentech, Inc. |
| 2003/11/6 | Coagulation factor XIII A-subunit (recombinant) | Prophylaxis of bleeding associated with congential factor XIII deficiency | TRETTEN | Routine prophylaxis of bleeding in patients with congenital Factor XIII A-subunit deficiency. | 2013/12/23 | Novo Nordisk, Inc. | |
| 2003/8/20 | Eculizumab | D03940 | Treatment of paroxysmal nocturnal hemoglobinuria | SOLIRIS | Treatment of paroxysmal nocturnal hemoglobinuria to reduce hemolysis | 2007/3/16/ 2007/6/20 (EU) |
Alexion Pharmaceuticals, Inc. Alexion Europe SAS (EU) |
| 2003/8/20 | Tinidazole | D01426 | Treatment of amebiasis | TINDAMAX | Treatment of intestinal amebiasis and amebic liver abcess caused by E. histolytica in both adults and pediatric patients older than three years of age. It is not indicated for the treatment of asymptomatic cyst passage. | 2004/5/17 | Presutti Laboratories, Inc. |
| 2003/8/19 | Glucarpidase | D10260 | Treatment of patients at risk of methotrexate toxicity | VORAXAZE | Treatment of toxic (>1 micromole/liter) plasma methotrexate concentrations in patients with delayed methotrexate clearance due to impaired renal function. | 2012/1/17 | BTG International Inc. |
| 2003/7/18 | Cholic acid | D10699 | Treatment of inborn errors of cholesterol and bile acid synthesis and metabolism | CHOLBAM KOLBAM, ORPHACOL (EU) |
Treatment of bile acid synthesis disorders due to single enzyme defects | 2015/3/17 2015/11/20, 2013/9/12 (EU) |
Retrophin, Inc. Retrophin Europe Ltd (EU), Laboratoires CTRS (EU) |
| 2003/7/10 | Plerixafor | D08971 | For use to improve the yield of progentor cells in the apheresis product for subsequest stem cell transplantation following myelosuppressive or myeloablative chemotherapy | MOZOBIL | To enhance mobilization of hematopoietic stem cells to the peripheral blood for collection and subsequent autologous transplantation in patients with lymphoma and multiple myeloma | 2008/12/15 2009/7/31 (EU) |
Genzyme Corporation Genzyme Europe B.V. (EU) |
| 2003/5/21 | Defibrotide | D07423 | For the treatment of hepatic veno-occlusive disease | DEFITELIO | For the treatment of adult and pediatric patients with hepatic veno-occlusive disease (VOD), also known as sinusoidal obstruction syndrome (SOS), with renal or pulmonary dysfunction following hematopoietic stem cell transplantation (HSCT). | 2016/3/30 | Jazz Pharmaceuticals, Inc. |
| 2003/5/21 | Repository corticotropin or adrenocorticotropic hormone | D06544 | Treatment of infantile spasms | H.P. ACTHAR GEL | To treat infantile spasms | 2010/10/15 | Questcor Pharmaceuticals, Inc. |
| 2003/5/12 | Cinacalcet | D03505 | Treatment of hypercalcemia in patients with parathyroid carcinoma | SENSIPAR | Treatment of hypercalcemia in patients with parathyroid carcinoma | 2004/3/8 | Amgen, Inc. |
| 2003/5/1 | Iron(III)-hexacyanoferrate(II) | D09736 | Treatment of patients with known or suspected internal contamination with radioactive or non-radioactive cesium or thallium | RADIOGARDASE | Treatment of patients with known or suspected internal contamination with radioactive cesium and/or radioactive or non-radioactive thallium to increase their rates of elimination | 2003/10/2 | Heyl Chemisch-Pharmzeutische Fabrik GMBH & Co, KG |
| 2003/4/4 | Ribavirin | D00423 | Treatment of chronic hepatitis C in pediatric patients | REBETOL | Treatment of chronic hepatitis C among previously untreated pediatric patients at least three years of age or older. | 2003/7/29 | Schering Corporation |
| 2003/3/27 | Romiplostim | D08990 | Treatment of immune thrombocytopenic purpura | NPLATE | Treatment of thrombocytopenia in patients with chronic immune (idiopathic) thrombocytopenic purpura (ITP) who have had an insufficient response to corticosteroids, immunoglobulins, or splenectomy | 2008/8/22 2009/2/4 (EU) |
Amgen, Inc. Amgen Europe B.V. (EU) |
| 2003/2/4 | Ecallantide | D03931 | Treatment of angioedema | KALBITOR | Treatment of acute attacks of hereditary angioedema (HAE) in patients 12 years of age and older | 2014/3/28 | Dyax Corp. |
| 2003/2/4 | Ecallantide | D03931 | Treatment of angioedema | KALBITOR | Treatment of acute attacks of hereditary angioedema in patients 16 years of age and older | 2009/12/1 | Dyax Corp. |
| 2003/1/15 | Bortezomib | D03150 | Treatment of multiple myeloma | VELCADE | First-line therapy of multiple myeloma. | 2008/6/20 | Millennium Pharmaceuticals, Inc. |
| 2003/1/15 | Bortezomib | D03150 | Treatment of multiple myeloma | VELCADE | Treatment of multiple myeloma patients who have received at least two prior therapies and have demonstrated disease progression on the last therapy | 2003/5/13 | Millennium Pharmaceuticals, Inc. |
| 2003/1/15 | Bortezomib | D03150 | Treatment of multiple myeloma | Velcade | Treatment of multiple myeloma patients who have received at least one prior therapy | 2005/3/25 | Millennium Pharmaceuticals, Inc. |
| 2003/1/15 | Pomalidomide | D08976 | Treatment of multiple myeloma | POMALYST IMNOVID (EU) |
Treatment of patients with multiple myeloma who have received at least two prior therapies including lenalidomide and bortezomib and have demonstrated disease progression on or within 60 days of completion of the last therapy. | 2013/2/8 2013/5/8 (EU) |
Celgene Corporation Celgene Europe Ltd (EU) |
| 2002/11/22 | Meloxicam | D00969 | Treatment of juvenile rheumatoid arthritis | MOBIC | For relief of the signs and symptoms of pauciarticular or polyarticular course Juvenile Rheumatoid Arthritis in patients 2 years of age or older | 2005/8/11 | Boehringer Ingelheim Pharmaceuticals, Inc. |
| 2002/11/21 | Deferasirox | D03669 | Treatment of chronic iron overload in patients with transfusion-dependent anemias | EXJADE | Treatment of chronic iron overload due to blood transfusions (transfusional hemosiderosis) in patients 2 years of age or older | 2005/11/2 2006/8/28 (EU) |
Novartis Pharmaceuticals Corporation Novartis Europharm Limited (EU) |
| 2002/5/17 | Mecasermin rinfabate | D04870 | Treatment of growth hormone insensitivity syndrome (GHIS) | IPLEX | Treatment of growth failure in children with severe primary IGF-1 deficiency (Primary IGFD) or with growth hormone (GH) gene deletion who have developed neutralizing antibodies to growth hormone | 2005/12/12 | Insmed, Inc. |
| 2002/4/18 | Tinidazole | D01426 | Treatment of giardiasis | TINDAMAX | Treatment of giardiasis caused by G. duodenalis (also termed G. lamblia) in both adults and pediatric patients older than three years of age | 2004/5/17 | Presutti Laboratories, Inc. |
| 2002/3/12 | Aztreonam | D00240 | Inhalation therapy for control of gram-negative bacteria in the respiratory tract of patients with cystic fibrosis | CAYSTON | To improve respiratory symptoms in cystic fibrosis (CF) patients with Pseudomonas aeruginosa | 2010/2/22 | Gilead Sciences (formerly Corus Pharma) |
| 2002/2/14 | Nitazoxanide | D02486 | Treatment of intestinal giardiasis | ALINIA | Treatment of diarrhea caused by Cryptosporidium parvum and Giardia lamblia | 2002/11/22 | Romark Laboratories, L.C. |
| 2002/2/7 | Clofarabine | D03546 | Treatment of acute lymphoblastic leukemia | CLOLAR EVOLTRA (EU) |
Treatment of pediatric patients 1 to 21 years old with relapsed or refractory acute lymphoblastic leukemia after at least two prior regimens | 2004/12/28 2006/5/29 (EU) |
Genzyme Corporation Genzyme Europe B.V. (EU) |
| 2001/12/12 | Deferiprone | D07416 | Treatment of iron overload in patients with hematologic disorders requiring chronic transfusion therapy | FERRIPROX | Treatment of patients with transfusional iron overload due to thalassemia syndromes when current chelation therapy is inadequate | 2011/10/14 | ApoPharma, Inc. A Division of |
| 2001/11/28 | Idursulfase | D04499 | Long term enzyme replacement therapy for patients with mucopolysaccharidosis II (Hunter Syndrome) | ELAPRASE | Indicated for patients with Hunter syndrome (mucopolysaccharidosis II, MPS II). Idursulfase has been shown to improve walking capacity in these patients | 2006/7/24/ 2007/1/8 (EU) |
Shire Human Genetic Therapies, Inc. Shire Human Genetic Therapies AB (EU) |
| 2001/11/1 | Imatinib mesylate | D01441 | Treatment of gastrointestinal stromal tumors | GLEEVEC | Adjuvant treatment of adult patients following complete resection Kit (CD 117) positive gastrointestinal stromal tumors (GIST). | 2008/12/19 | Novartis Pharmaceuticals Corp. |
| 2001/11/1 | Imatinib mesylate | D01441 | Treatment of gastrointestinal stromal tumors | GLEEVEC | Treatment of patients with Kit (CD117) positive unresectable and/or metastatic malignant gastrointestinal stromal tumors (GIST) | 2002/2/1 | Novartis Pharmaceuticals Corp. |
| 2001/10/19 | Porfimer | D03327 | For the ablation of High-Grade Dysplasia in Barrett's Esophagus in patients who are not considered to be candidates for esophagectomy | PHOTOFRIN | For the ablation of high-grade dysplasia in Barrett's esophagus patients who do not undergo esophagectomy | 2003/8/1 | Axcan Scandipharm Inc. |
| 2001/9/20 | Lenalidomide | D04687 | Treatment of multiple myeloma | REVLIMID | For use in combination with dexamethasone for the treatment of multiple myeloma patients who have received at least one prior therapy | 2006/6/29 2007/6/14 (EU) |
Celgene Corporation Celgene Europe Ltd (EU) |
| 2001/9/20 | Lenalidomide | D04687 | Treatment of multiple myeloma | REVLIMID | For use in combination with dexamethasone for the treatment of patients with multiple myeloma who have not received at least one prior therapy (first line treatment) | 2006/6/29 2007/6/14 (EU) |
Celgene Corporation Celgene Europe Ltd (EU) |
| 2001/12/3 | Azacitidine | D03021 | Treatament of myelodysplastic syndromes | VIDAZA | Treatment of patients with the following myelodysplastic syndrome subtypes: refractory anemia or refractory anemia with ringed sideroblasts (if accompanied by neutropenia or thrombocytopenia and requiring transfusions), refractory anemia with excess blasts, refractory anemia with excess blasts in transformation, and chronic myelomonocytic leukemia | 2004/5/19 2008/12/17 (EU) |
Celgene Corporation Celgene Europe Ltd (EU) |
| 2001/10/19 | Acetylcysteine | D00221 | For the intravenous treatment of moderate to severe acetaminophen overdose | ACETADOTE | For the use of Acetadote(registered TM) Injection, administered intravenously within 8 to 10 hours after ingestion of a potentially heptotoxic quantity of acetaminophen, to prevent or lessen hepatic injur | 2004/1/23 | Cumberland Pharmaceuticals, Inc. |
| 2001/8/28 | Pemetrexed disodium | D03828 | Treatment of malignant pleural mesothelioma | ALIMTA | Treatment of patients with malignant pleural mesothelioma whose disease is either unresectable or who are otherwise not candidates for curative surgery | 2004/2/4 | Eli Lilly and Company |
| 2001/8/22 | Metreleptin | D05014 | Treatment of metabolic disorders secondary to lipodystrophy | MYALEPT | Adjunct to diet as replacement therapy to treat the complications of leptin deficiency in patients with congenital or acquired generalized lipodystrophy. | 2014/2/24 | Aegerion Pharmaceuticals, Inc. |
| 2001/8/3 | Thyrotropin alfa | D06120 | Treatment of well-differentiated papillary, follicular or combined papillary/follicular carcinomas of the thyroid | THYROGEN | For use as an adjunctive treatment for radioiodine ablation of thyroid tissue remnants in patients who have undergone thyroidectomy for well-differentiated thyroid cancer and who do not have evidence of metastatic thyroid cancer | 2007/12/14 | Genzyme Corporation |
| 2001/2/21 | Pegloticase | D09316 | To control the clinical consequences of hyperuricemia in patients with severe gout in whom conventional therapy is contraindicated or has been ineffective. | KRYSTEXXA | Treatment of chronic gout in adult patients refractory to conventional treatment | 2010/9/14 | Crealta Pharmaceuticals LLC |
| 2001/1/31 | Imatinib | D01441 | Treatment of chronic myelogenous leukemia | GLEEVEC | Treatment of patients with chronic myeloid leukemia (CML) in blast crisis, accelerated phase, or in chronic phase after failure of interferon-alpha therapy | 2001/5/10 | Novartis Pharmaceuticals Corporation |
| 2000/12/27 | Somatropin [rDNA] | D02691 | Treatment of growth failure in children who were born small for gestational age. | GENOTROPIN | For long-term treatment of growth failure in children born small for gestational age who fail to manifest catch-up growth by two years of age. | 2001/7/25 | Pharmacia and Upjohn Company |
| 2000/10/11 | Rasburicase | D05704 | Treatment of malignancy-associated or chemotherapy-induced hyperuricemia. | ELITEK | Initial management of plasma uric acid levels in pediatric patients with leukemia,lymphoma, and solid tumor malignancies who are receiving anti-cancer therapy expected to result in tumor lysis and subsequent elevation of plasma uric acid. | 2002/7/12 | Sanofi-Synthelabo Research |
| 2000/10/6 | Bosentan | D01227 | Treatment of pulmonary arterial hypertension. | TRACLEER | Treatment of pulmonary arterial hypertension. | 2001/11/20 | Actelion Life Sciences Ltd. |
| 2000/9/11 | Lanreotide | D04666 | Treatment for acromegly | SOMATULINE DEPOT | Long-term treatment of acromegalic patients who have had an inadequate response to or cannot be treated with surgery and/or radiotherapy | 2007/8/30 | IPSEN, Inc. |
| 2000/8/18 | Zoledronate | D01968 | Treatment of tumor induced hypercalcemia. | ZOMETA, ZABEL | Treatment of hypercalcemia of malignancy. | 2001/8/20 | Novartis Pharmaceuticals Corp. |
| 2000/7/31 | Fluocinolone | D01825 | Treatment uveitis involving the posterior segment of the eye. | RETISERT | Treatment of chronic non-infectious uveitis affecting the posterior segment of the eye | 2005/4/8 | Bausch & Lomb Pharmaceuticals, Inc. |
| 2000/7/3 | Cetuximab | D03455 | Treatment of squamous cell cancer of the head and neck in patients who express epidermal growth factor receptor | ERBITUX | For use in combination with radiation therapy, for the treatment of locally or regionally advanced squamous cell carcinoma of the head and neck (SCCHN) and for use as a single agent for the treatment of patients with recurrent or metastatic SCCHN for whom prior platinum-based therapy has failed | 2006/3/1 | ImClone Systems Incorporated |
| 2000/6/29 | Teduglutide [rDNA origin] | D06053 | Treatment of short bowel syndrome. | GATTEX REVESTIVE (EU) |
Treatment of adult patients with short bowel syndrome (SBS) who are dependent on parenteral support | 2012/12/21 2012/8/30 (EU) |
NPS Pharmaceuticals, Inc. NPS Pharma Holdings Limited (EU) |
| 2000/6/12 | Centruroides immune F(ab)2 | Treatment of scorpion envenomations requiring medical attention. | ANASCORP | Treatment of clinical signs of scorpion envenomation | 2011/8/4 | Rare Disease Therapeutics, Inc. | |
| 2000/6/12 | Vigabatrin | D00535 | Treatment of infantile spasms. | SABRIL | For infantile spasms (IS) - 1 month to 2 years of age. | 2009/8/21 | H. Lundbeck A/S |
| 2000/3/7 | Synthetic porcine secretin | D02021 | For use in conjunction with diagnostic, therapeutic, or combined diagnostic/therapeutic procedures for pancreatic disorders to increase pancreatic fluid secretion. | SECREFLO | Use in secretin stimulation testing for stimulation of pancreatic secretions, including bicarbonate, to aid in the diagnosis of pancreatic exocrine dysfunction | 2002/4/4 | ChiRhoClin, Inc. |
| 2000/1/18 | Levodopa and carbidopa | D00253 | Treatment of late stage Parkinson's disease | DUODOPA | Treatment of motor flucuations in patients with advanced Parkinson's disease | 2015/1/9 | AbbVie, Inc. |
| 1999/11/24 | Gemtuzumab ozogamicin | D03259 | Treatment of CD33-positive acute myeloid leukemia. | MYLOTARG | Treatment of patients with CD33 positive acute myeloid leukemia in first relapse who are 60 years of age or older and who are not considered candidates for cytotoxic chemotherapy | 2000/5/17 | Wyeth-Ayerst Laboratories |
| 1999/10/28 | Somatropin (rDNA origin) | D02691 | Long-term treatment of children who have growth failure due to a lack of adequate endogenous growth hormone secretion. | NUTROPIN DEPOT | Long-term treatment of growth failure due to a lack of adequate endogenous growth hormone secetion for once- or twice-a-month administration. | 1999/12/22 | Genentech, Inc. |
| 1999/10/20 | Botulinum toxin type A | D00783 | Treatment of dynamic muscle contractures in pediatric cerebral palsy patients | DYSPORT | Treatment of lower limb spasticity in pediatric cerebral patients 2 years of age and older | 2016/7/29 | Ipsen Limited |
| 1999/9/14 | Epirubicin | D02214 | Treatment of breast cancer. | ELLENCE | Component of adjuvant therapy in patients with evidence of axillary node tumor involvement following resection of primary breast cancer. | 1999/9/15 | Pharmacia & Upjohn Company |
| 1999/8/13 | Mitoxantrone | D02166 | Treatment of secondary-progressive multiple sclerosis. | NOVANTRONE | Reducing neurologic disability and/or the frequency of clinical relapses in patients with secondary (chronic) progressive, progressive relapsing, or worsening relapsing-remitting multiple sclerosis (i.e., patients whose neurologic status is significantly adnormal between relapses). | 2000/10/13 | Serono, Inc. |
| 1999/8/13 | Mitoxantrone | D02166 | Treatment of progressive-relapsing multiple sclerosis. | NOVANTRONE | Reducing neurologic disability and/or the frequency of clinical relapses in patients with secondary (chronic) progressive, progressive relapsing, or worsening relapsing-remitting multiple sclerosis (i.e., patients whose neurologic status is significantly adnormal between relapses). | 2000/10/13 | Serono, Inc. |
| 1999/7/6 | Somatropin [rDNA] | D02691 | Treatment of short stature in patients with Prader-Willi syndrome. | GENOTROPIN | Long-term treatment of pediatric patients who have growth failure due to Prader-Willi syndrome (PWS). | 2000/6/20 | Pharmacia & Upjohn |
| 1999/6/18 | Bexarotene | D03106 | Treatment of cutaneous T-cell lymphoma. | TARGRETIN | Treatment of cutaneous manifestations of cutaneous T-cell lymphoma in patients who are refractory to at least one prior systemic therapy. | 1999/12/29 | Eisai, Inc. |
| 1999/6/18 | Synthetic porcine secretin | D02021 | For use in the evaluation of exocrine pancreas function. | SECREFLO | For use in secretin stimulation testing for: Stimulation of pancreatic secretions to facilitate the identification of the ampulla of Vater and accessory papilla during endoscopic retrograde cholangio-pancreatography (ERCP) | 2002/11/1 | ChiRhoClin, Inc. |
| 1999/6/18 | Synthetic porcine secretin | D02021 | For use in the diagnosis of gastrinoma associated with Zollinger-Ellison syndrome. | SECREFLO | Use in secretin stimulation testing for stimulation of gastrin secretion to aid in the diagnosis of gastrinoma | 2002/4/4 | ChiRhoClin, Inc. |
| 1999/3/22 | Epoprostenol | D01337 | Treatment of secondary pulmonary hypertension due to intrinsic precapillary pulmonary vascular disease. | FLOLAN | treatment of pulmonary arterial hypertension (WHO Group 1) to improve exercise capacity. Studies establishing effectiveness included predominantly patients with NYHA Functional Class III-IV symptoms and etiologies of idiopathic or heritable PAH or PAH associated with connective tissue diseases. | 2000/4/14 | GlaxoSmithKline |
| 1999/3/8 | Decitabine | D03665 | Treatment of myelodysplastic syndromes. | DACOGEN | for treatment of patients with myelodysplastic syndromes (MDS) including previously treated and untreated, de novo and secondary MDS of all French-American-British subtypes (refractory anemia, refractory anemia with ringed sideroblasts, refractory anemia with excess blasts, refractory anemia with excess blasts in transformation, and chronic myelomonocytic leukemia) and intermediate-1, intermediate-2, and high-risk International Prognostic Scoring System groups | 2006/5/2 2012/9/20 (EU) |
Otsuka Pharmaceutical Development Janssen-Cilag International N.V. (EU) |
| 1999/2/23 | C1-esterase inhibitor (recombinant) | Treatment of (acute attacks of) angioedema caused by hereditary or acquired C1-esterase inhibitor deficiency. | RUCONEST | Treatment of acute attacks of hereditary angioedema (HAE) in adult and adolescent patients. | 2014/7/16 | Santarus, Inc. | |
| 1999/2/17 | N-acetylgalactosamine-4-sulfatase, recombinant human | D06565 | Treatment of mucopolysaccharidosis Type VI (Maroteaux-Lamy syndrome). | NAGLAZYME | For patients with mucopolysaccharidosis VI. Galsulfase has been shown to improve walking and stair-climbing capacity. | 2005/5/31 2006/1/24 (EU) |
BioMarin Pharmaceutical, Inc. BioMarin Europe Ltd. (EU) |
| 1998/12/21 | Follitropin alfa, recombinant | D06400 | For the initiation and re-initiation of spermatogenesis in adult males with reproductive failure due to hypothalamic or pituitary dysfunction, hypogonadotropic hypogonadism. AMENDED indication 6/27/00: For the induction of spermatogenesis in men with primary and secondary hypogonadotropic hypogonadism in whom the cause of infertility is not due to primary testicular failure. | GONAL-F | For the induction of spermatogenesis in men with primary and secondary hypogonadotropic hypogonadism in whom the cause of infertility is not due to primary testicular failure. | 2000/5/24 | EMD Serono, Inc. |
| 1998/11/4 | Doxorubicin liposome | D01275 | Treatment of ovarian cancer. | DOXIL | Treatment of metastatic carcinoma of the ovary in patients with disease that is refractory to both paclitaxel- and platinium-based chemotherapy regimens. Refractory disease is defined as disease that has progressed while on treatment, or within 6 months of completing treatment. | 1999/6/28 | Alza Corporation |
| 1998/10/27 | Etanercept | D00742 | Reduction in signs and symptoms of moderately to severely active polyarticular-course juvenile rheumatoid arthritis in patients who have had an inadequate response to one or more disease-modifying anti-rheumatic drugs. | ENBREL | Reducing signs and symptoms of moderately to severely active polyarticular juvenile idiopathic arthritis (JIA) in patients ages 2 and older | 1999/5/27 | Immunex Corporation |
| 1998/10/14 | Thalidomide | D00754 | Treatment of multiple myeloma | THALOMID THALIDOMIDE (EU) |
Treatment of patients with newly diagnosed multiple myeloma | 2006/5/25 2008/4/16 (EU) |
Celgene Corporation Celgene Europe Limited (EU) |
| 1998/10/5 | Temozolomide | D06067 | Treatment of recurrent malignant glioma. | TEMODAR | Treatment of adult patients with newly diagnosed glioblastoma multiforme concomitatly with radiotherapy and then as maintenance treatment | 2005/3/15 | Schering-Plough Research Institute |
| 1998/10/5 | Temozolomide | D06067 | Treatment of recurrent malignant glioma. | TEMODAR | Treatment of adult patients with refractory anaplastic astrocytoma, i.e., patients at first relapse who have experienced disease progression on a drug regimen containing a nitrosourea and procarbazine | 1999/8/11 | Schering-Plough Research Institute |
| 1998/9/1 | Dexamethasone intravitreal implant | D00292 | Treatment of non-infectious ocular inflammation of the posterior segment in patients with intermediate, posterior, and panuveitis | OZURDEX | For treatment of non-infectious uveitis affecting the posterior segment of the eye | 2010/9/24 | Allergan |
| 1998/8/24/ | Octreotide | D06495 | Treatment of severe diarrhea and flushing associated with malignant carcinoid tumors. | SANDOSTATIN LAR | Supression of severe diarrhea and flushing associated with malignant carcinoid syndrome. | 1998/11/25 | Novartis Pharmaceuticals Corporation |
| 1998/8/24 | Octreotide | D06495 | Treatment of acromegaly. | SANDOSTATIN LAR | Reduction of growth hormone and IGF-1 (somatomedin C) in acromegaly. | 1998/11/25 | Novartis Pharmaceuticals Corporation |
| 1998/8/24 | Octreotide | D06495 | Treatment of diarrhea associated with vasoactive intestinal peptide tumors (VIPoma). | SANDOSTATIN LAR | Treatment of profuse watery diarrhea associated with VIPoma. | 1998/11/25 | Novartis Pharmaceuticals Corporation |
| 1998/5/29 | Miglustat | D05032 | Treatment of Gaucher disease. | ZAVESCA | Treatment of mild to moderate Type I Gaucher disease in adults for whom enzyme replacement therapy is not a therapeutic option (e.g., due to constraints such as allergy, hypersensitivity, or poor venous access). | 2003/7/31 2002/11/20 (EU) |
Actelion Pharmaceuticals Ltd Actelion Ltd (EU) |
| 1998/5/12 | Amifostine | D00226 | Reduction of the incidence of moderate to severe xerostomia in patients undergoing post-operative radiation treatment for head and neck cancer. | ETHYOL | Reduction of the incidence of moderate to severe xerostomia in patients undergoing post-operative radiation treatment for head and neck cancer, where the radiation port includes a substantial portion of the parotid glands. | 1999/6/24 | Medimmune Oncology, Inc. |
| 1998/3/24 | Alitretinoin | D02815 | Treatment of AIDS-related Kaposi's sarcoma. | PANRETIN | Topical treatment of cutaneous lesions in patients with AIDS-related Kaposi's sarcoma. | 1999/2/2 | Eisai, Inc |
| 1998/3/3 | Arsenic trioxide | D02106 | Treatment of acute promyelocytic leukemia. | TRISENOX | For induction of remission and consolidation in patients with acute promyelocytic leukemia (APL) who are refractory to, or have relapsed from, retinoid and anthracycline chemotherapy, and whose APL is characterized by the presence of the t(15;17) translocation of PML/RAR-alpha gene expression. | 2000/9/25 | Cephalon |
| 1998/2/10 | Rifaximin | D02554 | Treatment of hepatic encephalopathy | NORMIX | Reduction in the risk of overt hepatic encephalopathy (HE) recurrence in patients greater than or equal to 18 years of age | 2010/3/24 | Salix Pharmaceuticals, Inc. |
| 1998/1/20 | Carglumic acid | D07130 | Treatment of N-acetylglutamate synthetase deficiency. | CARBAGLU | As an adjunctive therapy for the treatment of acute hyperammonemia due to the deficiency of the hepatic enzyme N-acetylglutamate synthase (NAGS) and as manitenance therapy for chronic hyperammonemia due to NAGS deficiency | 2010/3/18 2003/1/24 (EU) |
Orphan Europe SARL Orphan Europe S.A.R.L. (EU) |
| 1997/12/12 | Basiliximab | D03058 | Prophylaxis of solid organ rejection. | SIMULECT | Prophylaxis of acute organ rejection in patients receiving renal transplantation when used as part of an immunosuppressive regimen that includes cyclosporine and corticosteroids. | 1998/5/12 | Novartis Pharmaceuticals Corporation |
| 1997/12/11 | Tetrabenazine | D08575 | Treatment of Huntington's disease | XENAZINE | Treatment of chorea associated with Huntington's disease | 2008/8/15 | Prestwick Pharmaceuticals, Inc |
| 1997/10/20 | Alemtuzumab | D02802 | Treatment of chronic lymphocytic leukemia. | CAMPATH | The treatment of patients with B-cell chronic lymphocytic leukemia who have been treated with alkylating agents and who have failed fludarabine therapy. | 2001/5/7 | Genzyme Corporation |
| 1997/9/24 | Laronidase | D04670 | Treatment of patients with mucopolysaccharidosis-I. | ALDURAZYME | Treatment for patients with Hurler and Hurler-Scheie forms of Mucopolysaccharidosis I (MPS I) and for patients with the Scheie form who have moderate to severe symptoms | 2003/4/30 | BioMarin Pharmaceutical, Inc. |
| 1997/8/19 | Cysteamine hydrochloride | D03635 | Treatment of corneal cystine crystal accumulation in cystinosis patients. | CYSTARAN | Treatment of corneal cystine crystal accumulation in patients with cystinosis | 2012/10/2 | Sigma-Tau Pharmaceuticals, Inc. |
| 1997/8/19 | Recombinant human acid alpha-glucosidase; alglucosidase alfa | D03207 | Treatment of glycogen storage disease type II. | 1. MYOZYME 2. LUMIZYME | (Lumizyme) Patients with Pompe disease (acid alpha-glucosidase (GAA deficiency)). | 2014/8/1 2006/3/29 (EU) |
Genzyme Corporation Genzyme Europe B.V. (EU) |
| 1997/8/19 | Recombinant human acid alpha-glucosidase; alglucosidase alfa | D03207 | Treatment of glycogen storage disease type II. | 1. MYOZYME 2. LUMIZYME | Lumizyme for patients 8 years and older with late (non-infantile) onset Pompe disease (GAA deficiency) who do not have evidence of cardiac hypertrophy. The safety and efficacy of Lumizyme (alglucosidase alfa) have not been evaluated in controlled clinical trials in infantile-onset patients, or in late (non-infantile) onset patients less than 8 years of age | 2010/5/24 2006/3/29 (EU) |
Genzyme Corporation Genzyme Europe B.V. (EU) |
| 1997/8/19 | Recombinant human acid alpha-glucosidase; alglucosidase alfa | D03207 | Treatment of glycogen storage disease type II. | 1. MYOZYME 2. LUMIZYME | Myozyme for use in patients with Pompe disease (GAA deficiency). Alglucosidase alfa has been shown to improve ventilator-free survival in patients with infantile onset Pompe disease as compared to an untreated historical control, whereas use of Alphaglucosidase in patients with other forms of Pompe disease has not been adequately studied to assure safety and efficacy. | 2006/4/28 2006/3/29 (EU) |
Genzyme Corporation Genzyme Europe B.V. (EU) |
| 1997/7/18 | Icodextrin 7.5% with Electrolytes Peritoneal Dialysis Solution | D03266 | Treatment of those patients having end stage renal disease and requiring peritoneal dialysis treatment. | EXTRANEAL | For use as a single daily exchange for the long (8-16 hour) dwell during continuous ambulatory peritoneal dialysis (CAPD) or automated peritoneal dialysis (APD) for the management of chronic renal failure. | 2002/12/20 | Baxter Healthcare Corporation |
| 1997/6/24 | Pegvisomant | D05394 | Treatment of acromegaly. | SOMAVERT | Treatment of acromegaly in patients who have had an inadequate response to surgery and/or radiation therapy and/or other medical therapies, or for whom these therapies are not appropriate | 2003/3/25 2002/11/13 (EU) |
Sensus Corporation Pfizer Limited (EU) |
| 1997/6/4 | Treprostinil | D06213 | Treatment of pulmonary arterial hypertension. | REMODULIN | Treatment of pulmonary arterial hypertension. | 2002/5/21 | United Therapeutics Corp. |
| 1997/3/25 | Paclitaxel | D00491 | Treatment of AIDS-related Kaposi's sarcoma. | TAXOL | For the second line treatment of AIDS-related Kaposi's sarcoma. | 1997/8/4 | Bristol-Myers Squibb Pharmaceutical Research Institute |
| 1997/2/13 | Lepirudin | D06880 | Treatment of heparin-associated thrombocytopenia type II. | REFLUDAN | For anticoagulation in patients with heparin-induced thrombocytopenia and associated thromboembolic disease in order to prevent further thromboembolic complications. | 1998/3/6 | Hoechst Marion Roussel |
| 1996/12/17 | Oprelvekin | D05266 | Prevention of severe chemotherapy-induced thrombocytopenia. | NEUMEGA | 1997/11/25 | Genetics Institute, Inc. | |
| 1996/12/12 | Nitazoxanide | D02486 | Treatment of cryptosporidiosis. | ALINIA | Treatment of diarrhea caused by Cryptosporidium parvum and Giardia lamblia | 2002/11/22 | Romark Laboratories, L.C. |
| 1996/12/10 | Liposomal amphotericin B | D00203 | Treatment of cryptococcal meningitis. | AMBISOME | Treatment of disseminated cryptococcosis including meningitis. | 1997/8/11 | Fujisawa USA, Inc. |
| 1996/12/6 | Liposomal amphotericin B | D00203 | Treatment of visceral leishmaniasis. | AMBISOME | Treatment of visceral leishmaniasis. | 1997/8/11 | Fujisawa USA, Inc. |
| 1996/11/18 | Somatropin for injection | D02691 | As replacement therapy for growth hormone deficiency in adults after epiphyseal closure. | NUTROPIN | 1997/12/15 | Genentech, Inc. | |
| 1996/11/7 | Filgrastim | D03235 | Reduction in the duration of neutropenia, fever, antibiotic use, and hospitalization, following induction and consolidation treatment for acute myeloid leukemia. | NEUPOGEN | Reducing the time to neutrophil recovery and the duration of fever, following induction or consolidation chemotherapy treatment of adults with acute myeloid leukemia. | 1998/4/2 | Amgen, Inc. |
| 1996/10/29 | Ibuprofen lysine | D06606 | Treatment of patent ductus arteriosus | NEOPROFEN | For closure of a clinically significant patent ductus arteriosus in premature infants weighing between 500 and 1500 g, who are no more than 32 weeks gestational age when usual medical management (e.g., fluid restriction, diuretics, respiratory support, etc.) is ineffective | 2006/4/13 | Lundbeck, Inc. |
| 1996/9/30 | Interferon gamma-1b | D00747 | Delaying time to disease progression in patients with severe, malignant osteopetrosis. | ACTIMMUNE | Delaying time to disease progression in patients with severe, malignant osteopetrosis. | 2000/2/10 | Horizon Pharma Ireland Limited |
| 1996/9/10 | Aldesleukin | D00748 | Treatment of metastatic melanoma. | PROLEUKIN | Treatment of adults with metastatic melanoma. | 1998/1/9 | Chiron Corporation |
| 1996/8/21 | Denileukin diftitox | D03682 | Treatment of patients with cutaneous T-cell lymphoma | ONTAK | Treatment of patients with persistent or recurrent cutaneous T-cell lymphoma whose malignant cells express the CD25 component of the IL-2 receptor. | 1999/2/5 | Eisai, Inc. |
| 1996/8/21 | Mitoxantrone | D02166 | Treatment of hormone refractory prostate cancer. | NOVANTRONE | In combination with corticosteroids as initial chemotherapy for the treatment of patients with pain related to advanced hormone-refractory prostate cancer. | 1996/11/13 | Serono |
| 1996/5/23 | Collagenase clostridium histolyticum | D09596 | Treatment of advanced (involutional or residual stage) Dupuytren's disease. | XIAFLEX | Treatment of adult patients with Dupuytren's contracture with a palpable cord | 2010/2/2 | Auxilium Pharmaceuticals, Inc. |
| 1996/3/12 | Collagenase clostridium histolyticum | D09596 | Treatment of Peyronie's disease. | XIAFLEX | Treatment of adult men with Peyronie's disease with a palpable plaque and curvature deformity of at least 30 degrees at the start of therapy. | 2013/12/6 | Auxilium Pharmaceuticals, Inc. |
| 1996/2/8 | Antihemophilic factor (recombinant) | D08232 | For the control and prevention of hemorrhagic episodes and for surgical prophylaxis in patients with hemophilia A (congenital factor VIII deficiency or classic hemophilia). | REFACTO, XYNTHA | 2000/3/6 | Wyeth Pharmaceuticals, Inc. | |
| 1996/1/18 | Albendazole | D00134 | Treatment of neurocysticercosis due to Taenia solium as: 1) chemotherapy of parenchymal, subarachnoidal and racemose (cysts in spinal fluid) neurocysticercosis in symptomatic cases and 2) prophylaxis of epilepsy and other sequelae in asymptomatic neurocysticercosis. | ALBENZA | Treatment of parenchymal neurocysticercosis due to active lesions caused by larval forms of the pork tapeworm, Taenia solium. | 1996/6/11 | Amedra Pharmaceuticals LLC |
| 1996/1/17 | Albendazole | D00134 | Treatment of hydatid disease (cystic echinococcosis due to E. granulosus larvae or alveolar echinococcosis due to E. multilocularis larvae). | ALBENZA | Treatment of cystic hydatid disease of the liver, lung, and peritoneum, caused by the larval form of the dog tapeworm, Echinococcus granulosus. | 1996/6/11 | Amedra Pharmaceuticals LLC |
| 1996/1/5 | Antihemophilic factor (human) | D02958 | Treatment of von Willebrand's disease | ALPHANATE | For surgical and/or invasive procedures in patients with von Willebrand Disease (VWD) in whom desmopressin is either ineffective or contraindicated. It is not indicated for patients with severe VWD (Type 3) undergoing major surgery | 2007/1/31 | Grifols Biologicals Inc. |
| 1995/12/12 | Mecasermin | D03297 | Treatment of growth hormone insensitivity syndrome. | INCRELEX | Long-term treatment of growth failure in children with severe primary IGF-1 deficiency (Primary IGFD) or with growth hormone (GH) gene deletion who have developed neutralizing antibodies to growth hormone. | 2005/08/30 2007/8/3 (EU) |
Ipsen Biopharmaceuticals, Inc. Ipsen Pharma (EU) |
| 1995/11/14 | Infliximab | D02598 | Treatment of Crohn's disease | REMICADE | Treatment of moderately to severely active Crohn's disease for the reduction of the signs and symptoms, in patients who have an inadequate response to conventional therapies; and treatment of patients with fistulizing Crohn's disease for the reduction in the number of draining enterocutaneous fistula(s). | 1998/8/24 | Centocor, Inc. |
| 1995/10/24 | Lidocaine patch 5% | D00358 | For relief of allodynia (painful hypersensitivity), and chronic pain in postherpetic neuralgia. | LIDODERM | 1999/3/19 | Teikoku Pharma USA, Inc. | |
| 1995/9/18 | Sterile talc powder | D01084 | Treatment of malignant pleural effusion. | SCLEROSOL | Prevention of recurrence of malignant pleural effusions in symptomatic patients. | 1997/12/24 | Bryan Corporation |
| 1995/8/23 | Lamotrigine | D00354 | Treatment of Lennox-Gastaut syndrome. | LAMICTAL | Adjunctive treatment of Lennox-Gastaut syndrome in pediatric and adult patients. | 1998/8/24 | Glaxo Wellcome Research and Development |
| 1995/7/26 | Thalidomide | D00754 | Treatment of erythema nodosum leprosum. | THALOMID | Acute treatment of the cutaneous manifestations of moderate to severe erythema nodosum leprosum (ENL) and as maintenance therapy for prevention and suppression of the cutaneous manifestations of ENL recurrences. | 1998/7/16 | Celgene Corporation |
| 1995/7/17 | Filgrastim | D03235 | For use in the mobilization of peripheral blood progenitor cells for collection in patients who will receive myeloablative or myelosuppressive chemotherapy. | NEUPOGEN | For use in the mobilization of hematopoietic progenitor cells into the peripheral blood collection by leukapheresis. | 1995/12/28 | Amgen, Inc. |
| 1995/6/9 | Rifapentine | D00879 | Treatment of pulmonary tuberculosis. | PRIFTIN | 1998/6/22 | Hoechst Marion Roussel | |
| 1995/6/7 | Ganciclovir intravitreal implant | D00333 | Treatment of cytomegalovirus retinitis. | VITRASERT | Treatment of CMV retinitis in patients with acquired immunodeficiency syndrome. | 1996/3/4 | Bausch & Lomb Surgical, Chiron Vision Products |
| 1995/5/16 | Nitisinone | D05177 | Treatment of tyrosinemia type 1 | ORFADIN | Treatment of hereditary tyrosinemia type 1 in combination with dietary restriction of tyrosine and phenylalanine. | 2016/4/22 | Swedish Orphan Biovitrum AB |
| Adjunctive therapy to dietary restriction of tyrosine and phenylalanine in the treatment of hereditary tyrosinemia type 1 | 2002/1/18 | Swedish Orphan Biovitrum AB (PUBL) | |||||
| 1995/3/6 | Glutamine | D00015 | For use with human growth hormone in the treatment of short bowel syndrome (nutrient malabsorption from the gastrointestinal tract resulting from an inadequate absorptive surface). | NUTRESTORE | Treatment of short bowel syndrome in patients receiving specialized nutritional support when used in conjunction with a recombinant human growth hormone that is approved for this indication. | 2004/6/10 | Emmaus Medical, Inc. |
| 1995/3/6 | Sargramostim | D05803 | To reduce neutropenia and leukopenia and decrease the incidence of death due to infection in patients with acute myelogenous leukemia. | LEUKINE | Following induction chemotherapy in older adult patients with acute myelogenous leukemia to shorten time to neutrophil recovery and to reduce the incidence of severe and life-threatening infections and infections resulting in death. | 1995/9/15 | Immunex Corporation |
| 1995/3/6 | Somatropin (r-DNA) | D02691 | For use alone or in combination with glutamine in the treatment of short bowel syndrome. | ZORBTIVE | Treatment of short bowel syndrome in patients receiving specialized nutritional support | 2003/12/1 | EMD Serono, Inc. |
| 1994/11/7 | Oxybate | D05866 | Treatment of narcolepsy. | XYREM | Treatment of excessive daytime sleepiness in patients with narcolepsy | 2005/11/18 | Jazz Pharmaceuticals |
| 1994/11/7 | Oxybate | D05866 | Treatment of narcolepsy. | XYREM | Treatment of cataplexy associated with narcolepsy | 2002/7/17 | Jazz Pharmaceuticals |
| 1994/10/27 | Buprenorphine in combination with naloxone | D10250 | Treatment of opiate addiction in opiate users | SUBOXONE | Treatment of opioid dependence in patients 16 years of age or older | 2002/10/8 | Reckitt Benckiser Pharmaceuticals, Inc. |
| 1994/10/13 | Tobramycin for inhalation | D00063 | Treatment of bronchopulmonary infections of Pseudomonas aeruginosa in cystic fibrosis patients. | TOBI | Management of cystic fibrosis patients with P.aeruginosa. | 1997/12/22 2011/7/20 |
Novartis Pharmaceuticals Corp Novartis Europharm Ltd (EU) |
| 1994/10/7 | Recombinant human luteinizing hormone | D04824 | For use in association with recombinant human follicle stimulating hormone for the treatment of women with chronic anovulation due to hypogonadotropic hypogonadism. | LUVERIS | Luveris (lutropin alfa for injection), concomitantly administered with Gonal-f (follitropin alfa for injection), is indicated for stimulation of follicular development in infertile hypogonadotropic hypogonadal women with profound LH deficiency (LH (less than) 1.2 IU/L). | 2004/10/8 | EMD Serono, Inc. |
| 1994/10/3 | Coagulation Factor IX (recombinant) | D05201 | Treatment of hemophilia B. | BENEFIX | For the control and prevention of hemorrhagic episodes in patients with hemophilia B (congenital factor IX deficiency and Christmas disease), including control and prevention of bleeding in surgical settings. | 1997/2/11 | Genetics Institute, Inc. |
| 1994/9/6 | Ibritumomab tiuxetan | D04489 | Treatment of B-cell non-Hodgkin's lymphoma. | ZEVALIN | Treatment of patients with relapsed or refractory low-grade, follicular, or transformed B-cell non-Hodgkin's lymphoma, including patients with Rituximab (Rituxan?) refractory follicular non-Hodgkin?s lymphoma | 2002/2/19 | Spectrum Pharmaceuticals, Inc. |
| 1994/9/6 | Somatropin | D02691 | Treatment of adults with growth hormone deficiency. | GENOTROPIN | 1997/10/31 | Pharmacia & Upjohn | |
| 1994/8/24 | Buffered intrathecal electrolyte/dextrose injection | For use as a diluent in the intrathecal administration of methotrexate and cytarabine for the prevention or treatment meningeal leukemia and lymphocytic lymphoma | ELLIOTTS B | For the intrathecal administration of methotrexate and cytarabine for the prevention or treatment of meningeal leukemia and lymphocytic lymphoma. | 1996/9/27 | Lukare Medical, LLC | |
| 1994/7/28 | Busulfan | D00248 | As preparative therapy in the treatment of malignancies with bone marrow transplantation. | BUSULFEX | For use in combination with cyclophosphamide as a conditioning regimen prior to allogeneic hematopoietic progenitor cell transplantation for chronic myelogenous leukemia. | 1999/2/4 | Otsuka Pharmaceutical Company |
| 1994/6/15 | Buprenorphine hydrochloride | D00836 | Treatment of opiate addiction in opiate users. | SUBUTEX | Treatment of opioid dependence in patients 16 years of age or older | 2002/10/8 | Reckitt Benckiser Pharmaceuticals, Inc. |
| 1994/6/13 | Rituximab | D02994 | Treatment of non-Hodgkin's B-cell lymphoma | RITUXAN | 1997/11/26 | Genentech, Inc. | |
| 1994/5/23 | Valrubicin | D02697 | Treatment of carcinoma in situ of the urinary bladder. | VALSTAR | For intravesical therapy of BCG-refractory carcinoma in situ of the urinary bladder in patients for whom immediate cystectomy would be associated with unacceptable morbidity or mortality. | 1998/9/25 | Anthra Pharmaceuticals, Inc. |
| 1994/5/16 | Betaine | D03103 | Treatment of homocystinuria. | CYSTADANE | Treatment of homocystinuria to decrease elevated homocysteine blood levels. | 1996/10/25 2007/2/15 (EU) |
Jazz Pharmaceuticals Orphan Europe S.A.R.L. (EU) |
| 1994/5/16 | Tositumomab and iodine I 131 tositumomab | D08622 | Treatment of non-Hodgkin's B-cell lymphoma. | BEXXAR | Treatment of patients with CD20 positive, follicular, non-Hodgkin's lymphoma, with and without transformation, whose disease is refractory to Rituximab and has relapsed following chemotherapy | 2003/6/27 | GlaxoSmithKline LLC |
| 1994/3/16 | Amiodarone HCl | D00636 | For the acute treatment and prophylaxis of life-threatening ventricular tachycardia or ventricular fibrillation. | CORDARONE | For initiation of treatment and prophylaxis of frequently recurring ventricular fibrillation and hemodynamically unstable ventricular tachycardia in patients refractory to other therapy. | 1995/8/3 | Wyeth-Ayerst Laboratories |
| 1994/3/14 | Sulfadiazine | D00587 | For use in combination with pyrimethamine for the treatment of Toxoplasma gondii encephalitis in patients with and without AIDS. | Toxoplasmosis, as adjunctive with pyrimethamine | 1994/7/29 | Eon Labs Manufacturing, Inc. | |
| 1994/1/12 | Antivenin, crotalidae polyvalent immune Fab (ovine) | D02963 | Treatment of envenomations inflicted by North American crotalid snakes. | CROFAB | Treatment of minimal and moderate North American Crotalidae envenomation | 2000/10/2 | Protherics, Inc. |
| 1993/12/10 | Sacrosidase | D05782 | Treatment of congenital sucrase-isomaltase deficiency | SUCRAID | Oral replacement therapy of the genetically determined sucrase deficiency, which is part of congenital sucrease-isomaltase deficiency. | 1998/4/9 | QOL Medical, LLC |
| 1993/11/22 | Benzoate/phenylacetate | D10205 | Treatment of acute hyperammonemia and associated encephalopathy in patients with deficiencies in enzymes of the urea cycle. | AMMONUL | Adjunctive therapy in the treatment of acute hyperammonemia and associated encephalopathy in patients with deficiencies in enzymes of the urea cycle. | 2005/2/17 | Medicis Pharmaceutical Corp. |
| 1993/11/22 | Dodium phenylbutyrate | D05868 | Treatment of urea cycle disorders: carbamylphosphate synthetase deficiency, ornithine transcarbamylase deficiency, and arginiosuccinic acid synthetase deficiency. | BUPHENYL | Adjunctive therapy in the chronic managment of patients with urea cycle disorders involving deficiencies of carbamylphosphate synthetase, ornithine transcarbamylase, or argininosuccinic acid synthetase. | 1996/4/30 | Medicis Pharmaceutical Corp. |
| 1993/11/17 | Aprotinin | D02971 | For prophylactic use to reduce perioperative blood loss and the homologous blood transfusion requirement in patients undergoing cardiopulmonary bypass surgery in the course of repeat coronary artery bypass graft surgery, and in selected cases of primary coronary artery bypass graft surgery where the risk of bleeding is especially high (impaired hemostasis) or where transfusion is unavailable or unacceptable. | TRASYLOL | 1993/12/29 | Bayer Corporation | |
| 1993/11/9 | Rho (D) immune globulin intravenous (human) | D05723 | Treatment of immune thrombocytopenic purpura. | WINRHO SD | Treatment of adults and children with chronic and acute immune thrombocytopenic purpura. | 1995/3/24 | Rh Pharmaceuticals, Inc. |
| 1993/9/17 | Bleomycin sulfate | D02177 | Treatment of malignant pleural effusion. | BLENOXANE | Treatment of malignant pleural effusion. | 1996/2/20 | Bristol-Myers Squibb Pharmaceutical Research Institute |
| 1993/8/2 | Pulmonary surfactant replacement, porcine | D08402 | For the treatment and prevention of respiratory distress syndrome in premature infants. | CUROSURF | Treatment (rescue) of respiratory distress syndrome in premature infants. | 1999/11/18 | Chiesi USA, Inc. |
| 1993/6/22 | Nitric oxide | D00074 | Treatment of persistent pulmonary hypertension in the newborn. | INOMAX | In conjunction with ventilatory support and other appropriate agents, in the treatment of term and near-term (>34 weeks) neonates with hypoxic respiratory failure associated with clinical or echocardiographic evidence of pulmonary hypertension, where it improves oxygenation and reduces the need for extracorporeal membrane oxygenation. | 1999/12/23 | INO Therapeutics, Inc. |
| 1993/6/2 | Cytarabine liposomal | D00168 | Treatment of neoplastic meningitis. | DEPOCYT | Intrathecal treatment of lymphomatous meningitis. | 1999/4/1 | Pacira Pharmaceuticals, Inc. |
| 1993/5/14 | Daunorubicin citrate liposome injection | Treatment of patients with advanced HIV-associated Kaposi's sarcoma. | DAUNOXOME | First line cytotoxic therapy for advanced, HIV related Kaposi's sarcoma. | 1996/4/8 | NeXstar Pharmaceuticals, Inc. | |
| 1993/4/22 | Apomorphine HCl | D02004 | Treatment of the on-off fluctuations associated with late-stage Parkinson's disease. | APOKYN | For the acute, intermittent treatment of hypomobility, "off" episodes ("end-of-dose-wearing-off" and unpredictable :on/off" episodes) associated with advanced Parkinson's disease | 2004/4/20 | US WorldMeds, LLC |
| 1993/3/16 | Riluzole | D00775 | Treatment of amyotrophic lateral sclerosis. | RILUTEK | Treatment of patients with amyotrophic lateral sclerosis. Riluzole extends survival and/or time to tracheostomy. | 1995/12/12 | Rhone-Poulenc Rorer Pharmaceuticals, Inc. |
| 1993/3/15 | Modafinil | D01832 | Treatment of excessive daytime sleepiness in narcolepsy. | PROVIGIL | Improve wakefulness in patients with excessive daytime sleepiness associated with narcolepsy. | 1998/12/24 | Cephalon, Inc. |
| 1993/3/5 | Daclizumab | D03639 | Prevention of acute renal allograft rejection. | ZENAPAX | Prophylaxis of acute organ rejection in patients receiving renal transplants, to be used as a part of an immunosuppressive regimen that includes cyclosporine and corticosteroids. | 1997/12/10 | Hoffmann-La Roche, Inc. |
| 1993/2/18 | Immune globulin intravenous, human | D06458 | Infection prophylaxis in pediatric patients affected with the human immunodeficiency virus. | GAMIMUNE N | For prophylaxis of children with clinical or immunologic evidence of human immunodeficiency virus to decrease the frequency of serious and minor bacterial infections and the frequency of hospitalization, and to increase the time free of serious bacterial infections. | 1993/12/27 | Bayer Corporation |
| 1992/11/25 | Topiramate | D00537 | Treatment of Lennox-Gastaut syndrome. | TOPAMAX | As adjunctive therapy in patients two years and older with siezures associated with Lennox-Gastaut syndrome. | 2001/8/28 | Johnson & Johnson Pharmaceutical R & D, LLC |
| 1992/10/16 | Allopurinol sodium | D07564 | Management of patients with leukemia, lymphoma, and solid tumor malignancies who are receiving cancer therapy which causes elevations of serum and urinary uric acid levels and who cannot tolerate oral therapy. | ALOPRIM | Management of patients with leukemia, lymphoma, and solid tumor malignancies who are receiving cancer therapy which causes elevations of serum and urinary uric acid levels and who cannot tolerate oral therapy. | 1996/5/17 | Catalytica Pharmaceuticals, Inc |
| 1992/10/16 | Antihemophilic factor/von Willebrand factor complex (human), dried, pasteurized | Treatment of patients with von Willebrand's disease | HUMATE-P | (1) In adult patients for treatment and prevention of bleeding in hemophilia A (classic hemophilia) and (2) in adult and pediatric patients for treatment of spontaneous and trauma-induced bleeding episodes in severe von Willebrand disease, and in mild and moderate von Willebrand disease where use of desmopressin is known or suspected to be inadequate. | 1999/4/1 | CSL Behring | |
| 1992/10/16 | C1-esterase-inhibitor, human, pasteurized | Prevention and/or treatment of acute attacks of hereditary angioedema. | BERINERT | Treatment of acute attacks of hereditary angioedema. | 2009/10/8 | CSL Behring LLC | |
| 1992/6/23 | Protein C concentrate | D08796 | For replacement therapy in congenital protein C deficiency for the prevention and treatment of thrombosis, pulmonary emboli, and purpura fulminans. | CEPROTIN | Prevention and treatment of venous thrombosis and purpura fulminans | 2007/3/30 | Baxalta US, Inc. |
| 1992/2/28 | Pilocarpine HCl | D02200 | Treatment of xerostomia and keratoconjunctivitis sicca in Sjogren's syndrome patients. | SALAGEN | Treatment of dry mouth in patients with Sjogren's syndrome. | 1998/2/11 | MGI Pharma, Inc. |
| 1992/2/25 | Diazepam viscous solution for rectal administration | D00293 | For the management of selected, refractory, patients with epilepsy, on stable regimens of antiepileptic drugs (AEDs), who require intermittent use of diazepam to control bouts of increased seizure activity. | 1997/7/29 | Valeant Pharmaceuticals | ||
| 1992/2/24 | Melphalan | D08173 | Treatment of patients with multiple myeloma for whom oral therapy is inappropriate. | ALKERAN | For the palliative treatment of patients with multiple myeloma for whom oral melphalan is not appropriate. | 1992/11/18 | Glaxo Wellcome Inc. |
| 1992/2/24 | Thyrotropin alpha | D06120 | As an adjunct in the diagnosis of thyroid cancer. | THYROGEN | As an adjunctive diagnostic tool for serum thyroglobulin (Tg) testing with or without radioiodine imaging in the follow-up of patients with thyroid cancer. | 1998/11/30 | Genzyme Corporation |
| 1992/2/19 | Aminosalicylic acid | D00162 | Treatment of tuberculosis infections | PASER | Treatment of tuberculosis | 1994/06/30 | Jacobus Pharmaceutical Company |
| 1992/1/16 | Botulinum toxin type B | D02735 | Treatment of cervical dystonia. | MYOBLOC | Treatment of patients with cervical dystonia to reduce the severity of abnormal head position and neck pain associated with cervical dystonia. | 2000/12/8 | Soltice Neurosciences, LLC |
| 1991/12/17 | Dexrazoxane | D07807 | For the prevention of cardiomyopathy associated with doxorubicin administration. | ZINECARD | Cardiomyopathy associated with doxorubicin administration in women with metastatic breast cancer who have received a cumulative dose of 300mg/m2. | 1995/05/26 | Pharmacia & Upjohn |
| 1991/12/16 | Interferon beta-1a | D04554 | Treatment of multiple sclerosis. | AVONEX | Treatment of relapsing forms of multiple sclerosis to slow the accumulation of physical disability and decrease the frequency of clinical exacerbations. | 1996/5/17 | Biogen, Inc. |
| 1991/12/5 | Amphotericin B lipid complex | D00203 | Treatment of invasive fungal infections. | ABELCET | Treatment of invasive fungal infections in patients who are refractory to or intolerant of conventional amphotericin B therapy. | 1996/10/18 | Liposome Company, Inc. |
| 1991/11/15 | Somatropin for injection | D02691 | Treatment of AIDS-associated catabolism/weight loss. | SEROSTIM | Treatment of AIDS wasting or cachexia. | 1996/8/23 | EMD Serono, Inc. |
| 1991/11/5 | Imiglucerase | D03020 | Replacement therapy in patients with types I, II, and III Gaucher's disease. | CEREZYME | Enzyme replacement therapy in patients with type I Gaucher's disease. | 1994/5/23 | Genzyme Corporation |
| 1991/11/4 | Halofantrine | D02485 | Treatment of mild to moderate acute malaria caused by susceptible strains of P. falciparum and P. vivax. | HALFAN | Treatment of adults who can tolerate oral medication and who have mild to moderate malaria caused by Plasmodium falciparum or Plasmodium vivax. | 1992/7/24 | SmithKline Beecham Pharmaceuticals |
| 1991/10/16 | Lodoxamide tromethamine | D04762 | Treatment of vernal keratoconjunctivitis. | ALOMIDE | Treatment of ocular disorders referred to by the terms vernal keratoconjunctivitis, vernal conjunctivitis, vernal keratitis. | 1993/9/23 | Alcon Laboratories, Inc. |
| 1991/9/19 | Exemestane | D00963 | Treatment of advanced breast cancer in postmenopausal women whose disease has progressed following tamoxifen therapy. | AROMASIN | Treatment of advanced breast cancer in postmenopausal women whose disease has progressed following tamoxifen therapy. | 199910/21 | Pharmacia & Upjohn |
| 1991/9/19 | Toremifene | D00967 | Hormonal therapy of metastatic carcinoma of the breast. | FARESTON | Treatment of metastatic breast cancer in postmenopausal women with estrogen positive or receptor unknown tumors. | 1997/5/29 | Orion Corporation |
| 1991/8/14 | Atovaquone | D00236 | Prevention of Pneumocystis carinii pneumonia (PCP) in high-risk, HIV-infected patients defined by a history of one or more episodes of PCP and/or a peripheral CD4+ (T4 helper/inducer) lymphocyte count less than or equal to 200/mm3. | MEPRON | Prevention of PCP in patients who are intolerant to trimethoprim-sulfamethoxazole. | 1999/01/5 | Glaxo Wellcome Research and Development |
| 1991/8/1 | Levoleucovorin | D04715 | For use in conjunction with high-dose methotrexate in the treatment of osteosarcoma. | FUSILEV | Levoleucovorin rescue is indicated after high-dose methotrexate therapy in osteosarcoma. Levoleucovorin is also indicated to diminish the toxicity and counteract the effects of impaired methotrexate elimination and of inadvertent overdosage of folic acid antagonists | 2008/3/7 | Spectrum Pharmaceuticals, Inc. |
| 1991/7/1 | Epoetin alfa | D03231 | Treatment of anemia associated with HIV infection or HIV treatment. | EPOGEN | Treatment of AZT-induced anemia in HIV infected patients. | 1990/12/31 | Amgen, Inc. |
| 1991/6/20 | Ursodiol | D00734 | Treatment of patients with primary biliary cirrhosis | URSO 250 | 1997/12/10 | Aptalis Pharma US, Inc. | |
| 1991/6/4 | Fosphenytoin | D02096 | For the acute treatment of patients with status epilepticus of the grand mal type. | CEREBYX | For the control of generalized convulsive status epilepticus. | 1996/08/05 | Warner-Lambert Company |
| 1991/4/18 | Ofloxacin | D00453 | Treatment of bacterial corneal ulcers. | OCUFLOX | Treatment of bacterial corneal ulcers. | 1996/5/22 | Allergan, Inc. |
| 1991/1/25 | Cysteamine | D10468 | Treatment of nephropathic cystinosis. | CYSTAGON | Treatment of nephropathic cystinosis in adults and children. | 1994/8/15 | Mylan Laboratories, Inc. |
| 1991/1/22 | Desmopressin acetate | D02235 | Treatment of mild hemophilia A and von Willebrand's disease. | Treatment of patients with hemophilia A or von Willebrand's disease (type I) whose factor VIII coagulant activity level is greater than 5%. | 1994/3/7 | Aventis Behring L.L.C. | |
| 1991/1/16 | Dornase alfa | D03896 | To reduce mucous viscosity and enable the clearance of airway secretions in patients with cystic fibrosis. | PULMOZYME | In conjunction with standard therapies in the management of cystic fibrosis patients to reduce the frequency of respiratory infections requiring parenteral antibiotics and to improve pulmonary function. | 1993/12/30 | Genentech, Inc. |
| 1991/1/15 | Dronabinol | D00306 | For the stimulation of appetite and prevention of weight loss in patients with a confirmed diagnosis of AIDS. | MARINOL | Treatment of anorexia associated with weight loss in patients with AIDS. | 199212/22 | Unimed Pharmaceuticals, Inc. |
| 1990/12/18 | levoleucovorin | D04715 | For use in combination chemotherapy with the approved agent 5-fluorouracil in the palliative treatment of metastatic adenocarcinoma of the colon and rectum | FUSILEV | For use in combination chemotherapy with 5-fluorouracil in the palliative treatment of patients with advanced metastatic colorectal cancer | 2011/4/29 | Spectrum Pharmaceuticals, Inc. |
| 1990/11/15 | Cladribine | D01370 | Treatment of hairy cell leukemia. | LEUSTATIN | 1993/2/26 | R. W. Johnson Pharmaceutical Research Institute | |
| 1990/11/7 | Filgrastim | D03235 | Treatment of patients with severe chronic neutropenia (absolute neutrophil count less than 500/mm3). | NEUPOGEN | Treatment of patients with severe chronic neutropenia. | 1994/12/19 | Amgen, Inc. |
| 1990/10/24 | Tretinoin | D00094 | Treatment of acute promyelocytic leukemia. | VESANOID | Induction of remission in patients with acute promyelocytic leukemia who are refractory to or unable to tolerate anthracycline based cytotoxic chemotherapeutic regimens. | 1995/11/22 | Hoffmann-La Roche, Inc. |
| 1990/10/1 | Filgrastim | D03235 | Treatment of neutropenia associated with bone marrow transplants. | NEUPOGEN | Reduce the duration of neutropenia and neutropenia sequelae in patients with non-myeloid malignancies undergoing ablative chemotherapy followed by BMT. | 1994/6/15 | Amgen, Inc. |
| 1990/10/1 | Hydroxyurea | D00341 | Treatment of patients with sickle cell anemia as shown by the presence of hemoglobin S. | DROXIA SIKLOS (EU) |
To reduce the frequency of painful crises and to reduce the need for blood transfusions in adult patients with sickle cell anemia with recurrent moderate to severe painful crises (generally at least 3 during the preceding 12 months). | 1998/2/25 2007/6/29 (EU) |
Bristol-Myers Squibb Pharmaceutical Research Institute Addmedica (EU) |
| 1990/9/27 | Respiratory syncytial virus immune globulin (Human) | Prophylaxis of respiratory syncytial virus lower respiratory tract infections in infants and young children at high risk of RSV disease. | RESPIGAM | For the prevention of serious lower respiratory tract infections caused by RSV in children less than 24 months of age with bronchopulmonary dysplasia or a history of prematurity (less than or equal to 35 weeks gestation). | 1996/1/18 | MedImmune & Massachussetts Public Health Biologics Labs. | |
| 1990/9/24 | Pilocarpine | D02200 | Treatment of xerostomia induced by radiation therapy for head and neck cancer. | SALAGEN | Treatment of symptoms of xerostomia from salivary gland hypofunction caused by radiotherapy for cancer of the head and neck. | 1994/3/22 | MGI Pharma, Inc. |
| 1990/9/10 | Atovaquone | D00236 | Treatment of AIDS associated Pneumocystis Carinii Pneumonia. | MEPRON | For the acute oral treatment of mild to moderate Pneumocystis carinii pneumonia in patients who are intolerant to trimethoprim-sulfamethoxazole. | 1992/11/25 | Glaxo Wellcome Inc. |
| 1990/7/30 | Liothyronine sodium injection | D01011 | Treatment of myxedema coma/precoma. | TRIOSTAT | Treatment of myxedema coma/precoma. | 1991/12/31 | SmithKline Beecham Pharmaceuticals |
| 1990/7/18 | Mafenide acetate solution | D01166 | For use as an adjunctive topical antimicrobial agent to control bacterial infection when used under moist dressings over meshed autografts on excised burn wounds. | SULFAMYLON | 1998/6/5 | Mylan Laboratories, Inc. | |
| 1990/7/12 | Morphine sulfate concentrate (preservative free) | D00842 | For use in microinfusion devices for intraspinal administration in the treatment of intractable chronic pain. | INFUMORPH | For intrathecal or epidural infusion in the treatment of tractable chronic pain. It was developed for use in continuous microinfusion devices and may require dilution before use as directed. | 1991/7/19 | Elkins-Sinn, Inc. |
| 1990/7/5 | Coagulation Factor IX (human) | D08797 | For use as replacement therapy in patients with hemophilia B for the prevention and control of bleeding episodes, and during surgery to correct defective hemostasis. | ALPHANINE | 1990/12/31 | Alpha Therapeutic Corporation | |
| 1990/5/30 | Amifostine | D00226 | For use as a chemoprotective agent for cisplatin in the treatment of advanced ovarian carcinoma. | ETHYOL | To reduce the cumulative renal toxicity associated with repeated administration of cisplatin in patients with advanced ovarian cancer. | 1995/12/8 | Medimmune Oncology, Inc. |
| 1990/5/8 | Somatropin | D02691 | Treatment of short stature associated with Turner syndrome. | HUMATROPE | Treatment of short stature associated with Turner syndrome in patients whose epiphyses are not closed. | 1996/12/30 | Eli Lilly and Company |
| 1990/5/3 | Sargramostim | D05803 | Treatment of neutropenia associated with bone marrow transplant, for the treatment of graft failure and delay of engraftment, and for the promotion of early engraftment. | LEUKINE | 1991/3/5 | Immunex Corporation | |
| 1989/12/18 | Rifabutin | D00424 | Prevention of disseminated Mycobacterium avium complex disease in patients with advanced HIV infection. | MYCOBUTIN | Prevention of disseminated Mycobacterium avium complex disease in patients with advanced HIV infections. | 1992/12/23 | Adria Laboratories, Inc. |
| 1989/12/13 | polifeprosan 20 with carmustine | D00254 | Treatment of malignant glioma. | GLIADEL | As an adjunct to surgery to prolong survival in patients with recurrent glioblastoma multiforme for whom surgical resection is indicated | 1996/9/23 | Guilford Pharmaceuticals, Inc. |
| 1989/12/13 | polifeprosan 20 with carmustine | D00254 | Treatment of malignant glioma. | GLIADEL | Expanding the indication to include patients with malignant glioma undergoing primary surgical resection. | 2003/2/25 | Guilford Pharmaceuticals, Inc. |
| 1989/11/24 | Corticorelin ovine triflutate | D03592 | For use in differentiating pituitary and ectopic production of ACTH in patients with ACTH-dependent Cushings syndrome. | ACTHREL | To differentiate between pituitary and ectopic production of ACTH in patients with ACTH-dependent Cushing's syndrome. | 1996/5/23 | Ferring Laboratories, Inc. |
| 1989/10/20 | Colfosceril palmitate, cetyl alcohol, tyloxapol | 1. Prevention of hyaline membrane disease, also known as respiratory distress syndrome, in infants born at 32 weeks gestation or less 2. prevention of hyaline membrane disease (HMD), also known as respiratory distress syndrome (RDS), in infants born at 32 weeks gestation or less and in the treatment of established HMO at all gestational ages. | EXOSURF NEONATAL | Prevention of hyaline membrane disease (HMD), also known as respiratory distress syndrome (RDS), in infants born at 32 weeks gestation or less and in the treatment of established HMO at all gestational ages | 1990/8/2 | Glaxo Wellcome Inc. | |
| 1989/10/20 | Pegaspargase | D05387 | Treatment of acute lymphocytic leukemia. | ONCASPAR | Combination chemotherapy for the treatment of patients with acute lymphoblastic leukemia who are hypersensitive to native forms of L-asparaginase. | 1994/2/1 | Sigma-tau Pharmaceuticals, Inc. |
| 1989/9/25 | Antihemophilic factor (recombinant) | D05401 | Prophylaxis and treatment of bleeding in individuals with hemophilia A or for prophylaxis when surgery is required in individuals with hemophilia A. | KOGENATE | Treatment and prophylaxis of bleeding in patients with hemophilia A (not von Willebrand's disease) | 1993/2/25 | Bayer Corporation |
| 1989/9/25 | Satumomab pendetide | D04528 | Detection of ovarian carcinoma. | ONCOSCINT CR/OV | For determining the extent and location of extraphepatic malignant disease in patients with known colorectal and ovarian cancer. | 1992/12/29 | Cytogen Corporation |
| 1989/8/28/ | Citric acid, glucono-delta-lactone and magnesium carbonate | Treatment of renal and bladder calculi of the apatite or struvite variety. | RENACIDIN | 1990/10/2 | United-Guardian, Inc. | ||
| 1989/8/4 | Somatropin for injection | D02691 | Treatment of growth retardation associated with chronic renal failure. | NUTROPIN | Treatment of children with growth failure associated with chronic renal insufficency. | 1993/11/17 | Genentech, Inc. |
| 1989/6/27 | Coagulation factor IX | D08797 | Replacement treatment and prophylaxis of the hemorrhagic complications of hemophilia B. | MONONINE | Prevention and control of bleeding in factor IX deficiency, also known as hemophilia B or Christmas disease. | 1992/8/20 | Armour Pharmaceutical Company |
| 1989/6/6 | Interferon alfa-2a | D00745 | Treatment of chronic myelogenous leukemia. | ROFERON A | Treatment of chronic myelogenous leukemia. | 1995/10/19 | Hoffmann-La Roche, Inc. |
| 1989/6/6 | Porfimer sodium | D03327 | For the photodynamic therapy of patients with primary or recurrent obstructing (either partially or completely) esophageal carcinoma. | PHOTOFRIN | Photodynamic therapy with PHOTOFRIN for the palliation of patients with completely obstructing esophageal cancer, or of patients with partially obstructing esophageal cancer who cannot be satisfactorily treated with Nd:YAG laser therapy. | 1995/12/27 | QLT Phototherapeutics, Inc. |
| 1989/4/18 | Fludarabine phosphate | D01907 | Treatment of chronic lymphocytic leukemia (CLL), including refractory CLL. | FLUDARA | 1991/4/18 | Berlex Laboratories, Inc. | |
| 1989/3/23 | Somatropin for injection | D02691 | Treatment of short stature associated with Turner's syndrome. | NUTROPIN | Treatment of growth failure associated with Turner syndrome. | 1996/12/30 | Genentech, Inc. |
| 1989/1/31 | Botulism immune globulin | Treatment of infant botulism. | BABYBIG | Indicated for treatment of infant botulism caused by type A or type B Clostridium botulinum. | 2003/10/23 | California Department of Health Services | |
| 1989/1/24 | Clonidine | D00604 | For continous epidural administration as adjunctive therapy with intraspinal opiates for the treatment of pain in cancer patients tolerant to, or unresponsive to, intraspinal opiates. | DURACLON | In combination with opiates for the treatment of severe pain in cancer patients that is not adequately relieved by opioid analgesics alone. | 1996/10/2 | Roxane Laboratories, Inc. |
| 1989/1/24 | Felbamate | D00536 | Treatment of Lennox-Gastaut syndrome. | FELBATOL | As adjunctive therapy in the treatment of partial and generalized seizures associated with the Lennox-Gastaut syndrome in children. | 1993/7/29 | Wallace Laboratories |
| 1988/12/22 | Calcium acetate | D00931 | Treatment of hyperphosphatemia in end stage renal failure. | PHOS-LO | 1990/12/10 | Fresenius Medical Care North America | |
| 1988/12/22 | Fomepizole | D00707 | Treatment of methanol or ethylene glycol poisoning. | ANTIZOL | Use for suspected or confirmed methanol poisoning, either alone or in combination with hemodialysis | 2000/12/8 | Jazz Pharmaceuticals |
| 1988/12/22 | Fomepizole | D00707 | Treatment of methanol or ethylene glycol poisoning. | ANTIZOL | As an antidote to ethylene glycol (antifreeze) poisoning, or for use in suspected ethylene glycol ingestion. | 1997/12/4 | Jazz Pharmaceuticals |
| 1988/11/17 | Interferon beta-1b | D00746 | Treatment of multiple sclerosis. | BETASERON | In ambulatory patients with relapsing-remitting multiple sclerosis to reduce the frequency of clinical exacerbations. | 1993/7/23 | Chiron Corp. & Berlex Laboratories |
| 1988/9/30 | Interferon gamma 1-b | D00747 | Treatment of chronic granulomatous disease. | ACTIMMUNE | 1990/12/20 | Horizon Pharma Ireland Limited | |
| 1988/9/23 | Sotalol HCl | D01026 | 1. Treatment of life-threatening ventricular tachyarrhythmias 2. Prevention of life treatening ventricular tachyarrhythmias | BETAPACE | Treatment of life-threatening ventricular tachyarrhythmias | 1992/10/30 | Berlex Laboratories, Inc. |
| 1988/9/20 | Caffeine | D07603 | Treatment of apnea of prematurity. | CAFCIT PEYONA (EU) |
Short term treatment of apnea of prematurity in infants between 28 and less than 33 weeks gestational age. | 1999/9/21 2009/7/2 (EU) |
O.P.R. Development, L.P. Chiesi Farmaceutici SpA (EU) |
| 1988/9/14 | Aldesleukin | D00748 | Treatment of metastatic renal cell carcinoma. | PROLEUKIN | Treatment of adults (>18 years old) with metastatic renal cell carcinoma. | 1992/5/5 | Chiron Corporation |
| 1988/9/14 | Sermorelin acetate | D02117 | Treatment of idiopathic or organic growth hormone deficiency in children with growth failure. | GEREF | 1997/9/26 | EMD Serono, Inc. | |
| 1988/9/6/ | Levocarnitine | D02176 | Treatment of manifestations of carnitine deficiency in patients with end stage renal disease who require dialysis. | CARNITOR | Prevention and treatment of carnitine deficiency in patients with end stage renal disease who are undergoing dialysis. | 1999/12/15 | Sigma-Tau Pharmaceuticals, Inc. |
| 1988/8/17 | Leucovorin | D01211 | For rescue use after high dose methotrexate therapy in the treatment of osteosarcoma. | LEUCOVORIN CALCIUM | 1988/8/31 | Immunex Corporation | |
| 1988/8/10 | Histrelin acetate | D02116 | Treatment of central precocious puberty. | SUPPRELIN | Treatment of central precocious puberty. | 1991/12/24 | Roberts Pharmaceutical Corp. |
| 1988/7/25 | Idarubicin HCl for injection | D01747 | Treatment of acute myelogenous leukemia, also referred to as acute nonlymphocytic leukemia. | IDAMYCIN | 1990/9/27 | Adria Laboratories, Inc. | |
| 1988/7/25 | Leuprolide acetate | D00989 | Treatment of central precocious puberty | LUPRON | Treatment of children with central precocious puberty | 1993/4/16/ | Tap Pharmaceuticals, Inc. |
| 1988/7/20 | Nafarelin acetate | D00990 | Treatment of central precocious puberty. | SYNAREL | Treatment of central precocious puberty (gonadotropin-dependent precocious puberty) in children of both sexes. | 1992/2/26 | Syntex (USA), Inc. |
| 1988/6/28 | Zalcitabine | D00412 | Treatment of AIDS. | HIVID | Combination therapy with zidovudine is indicated for the treatment of adult patients with advanced HIV infection (CD4 count < 300) who have demonstrated malignant clinical or immunologic deterioration. | 1992/6/19 | Hoffmann-La Roche, Inc. |
| 1988/6/6 | Coagulation factor VIIa (recombinant) | D00172 | Treatment of bleeding episodes in hemophilia A or B patients with inhibitors to Factor VIII or Factor IX. | NOVOSEVEN | Treatment of bleeding episodes in hemophilia A or B patients with inhibitors to Factor VIII or Factor IX. | 1999/3/25 | Novo Nordisk, Inc. |
| 1988/4/13 | Mefloquine HCL | D00831 | For use in the treatment of acute malaria due to Plasmodium falciparum and Plasmodium vivax, and for the prophylaxis of Plasmodium falciparum malaria which is resistant to other available drugs | LARIAM | Treatment of acute malaria due to Plasmodium falciparum and Plasmodium vivax | 1989/5/2 | Hoffmann-La Roche, Inc. |
| 1988/4/13 | Mefloquine HCL | D00831 | For use in the treatment of acute malaria due to Plasmodium falciparum and Plasmodium vivax, and for the prophylaxis of Plasmodium falciparum malaria which is resistant to other available drugs | LARIAM | Prophylaxis of Plasmodium falciparum malaria which is resistant to other available drugs | 1989/5/3 | Hoffmann-La Roche, Inc. |
| 1988/4/13 | Megestrol acetate | D00952 | Treatment of patients with anorexia, cachexia, or significant weight loss (=/>10% of baseline body weight) and confirmed diagnosis of AIDS. | MEGACE | Treatment of anorexia, cachexia, or an unexplained significant weight loss in patients with a diagnosis of acquired immune deficiency syndrome. | 1993/9/10 | Bristol-Myers Squibb Pharmaceutical Research Institute |
| 1988/1/27 | Anagrelide | D02933 | Treatment of essential thrombocythemia. | AGRYLIN | Treatment of patients with essential thrombocythemia to reduce the elevated platelet count and the risk of thrombosis and to ameliorate associated symptoms. | 1997/3/14 | Roberts Pharmaceutical Corp. |
| 1988/1/19 | Ceramide trihexosidase/alpha-galactosidase A | D03228 | Treatment of Fabry's disease. | FABRAZYME | For use in patients with Fabry disease to reduce globotriaosylceramide (GL-3) deposition in capillary endothelium of the kidney and certain other cell types | 2003/4/24 | Genzyme Corporation |
| 1988/1/12 | Pentamidine isethionate | D00834 | Prevention of Pneumocystis carinii pneumonia in patients at high risk of developing this disease. | NEBUPENT | 1989/6/15 | Fujisawa USA, Inc. | |
| 1987/12/14 | Interferon alfa-2a (recombinant) | D00745 | Treatment of AIDS related Kaposi's sarcoma. | ROFERON-A | 1988/11/21 | Hoffmann-La Roche, Inc. | |
| 1987/11/25 | Urofollitropin | D06269 | For induction of ovulation in patients with polycystic ovarian disease who have an elevated LH/FSH ratio and who have failed to respond to adequate clomiphene citrate therapy. | METRODIN | 1986/9/18 | EMD Serono, Inc. | |
| 1987/11/10 | Baclofen | D00241 | Treatment of intractable spasticity caused by spinal cord injury, multiple sclerosis, and other spinal diseases (including spinal ischemia, spinal tumor, transverse myelitis, cervical spondylosis, and degenerative myelopathy). | LIORESAL | Management of severe spasticity of spinal cord origin in patients who are unresponsive to oral baclofen therapy or experience intolerable CNS side effects at effective doses. | 1992/6/25 | Medtronic, Inc. |
| 1987/11/9 | Glatiramer acetate | D04318 | Treatment of multiple sclerosis. | COPAXONE | For reduction of the frequency of relapses in patients with relapsing-remitting multiple sclerosis. | 1996/12/20 | Teva Pharmaceuticals USA |
| 1987/10/22 | Metronidazole (topical) | D00409 | Treatment of acne rosacea. | METROGEL | 1988/11/22 | Galderma Laboratories, Inc. | |
| 1987/9/10 | Pentostatin for injection | D00155 | Treatment of hairy cell leukemia. | NIPENT | Single agent treatment for adult patients with alpha-interferon-refractory hairy cell leukemia. | 1991/10/11 | SuperGen, Inc. |
| 1987/8/3 | Cytomegalovirus immune globulin (human) | Prevention or attenuation of primary cytomegalovirus disease in immunosuppressed recipients of organ transplants. | CYTOGAM | #1) Attenuation of primary CMV disease associated with kidney transplant recipients who are seronegative for CMV and who receive a kidney from a CMV seropositve donor; #2) Prophylaxis of CMV disease associated transplantation of kidney (all other), lung, liver, pancreas and heart. | 1998/12/4 | CSL Behring LLC | |
| 1987/7/13 | Mitoxantrone HCl | D02166 | Treatment of acute myelogenous leukemia, also referred to as acute nonlymphocytic leukemia. | NOVANTRONE | 1987/12/23 | Lederle Laboratories | |
| 1987/7/10 | Somatropin (rDNA origin) injection | D02691 | 1. Treatment of growth failure in children due to inadequate growth hormone secretion 2. Treatment of short stature associated with Turner's syndrome | NORDITROPIN | Long-term treatment of children who have growth failure due to inadequate secretion of endogenous growth hormone. | 2000/6/20 | Novo Nordisk Pharmaceuticals |
| 1987/6/24 | Interferon alfa-2b (recombinant) | D02745 | Treatment of AIDS-related Kaposi's sarcoma. | INTRON A | Treatment of selected patients with AIDS-related Kaposi's sarcoma. | 1988/11/21 | Schering Corporation |
| 1987/6/2 | Dalfampridine | D04127 | Relief of symptoms of multiple sclerosis | AMPYRA | Treatment to improve walking in patients with multiple sclerosis | 2010/01/22 | Acorda Therapeutics |
| 1987/4/22 | Gonadorelin acetate | D04361 | For induction of ovulation in women with hypothalamic amenorrhea due to a deficiency or absence in the quantity or pulse pattern of endogenous GnRH secretion. | LUTREPULSE | 1989/10/10 | Ferring Laboratories, Inc. | |
| 1987/3/6 | Somatropin (rDNA origin) | D02691 | Treatment of idiopathic or organic growth hormone deficiency in children with growth failure. | SAIZEN | Long term treatment of children with growth failure due to inadequate secretion of endogenous growth hormone. | 1996/10/8 | EMD Serono, Inc. |
| 1987/3/6 | Somatropin | D02691 | For use in the long-term treatment of children who have growth failure due to a lack of adequate endogenous growth hormone secretion. | NUTROPIN | 1985/10/17 | Genentech, Inc. | |
| 1987/1/20 | Calcitonin-human for injection | Treatment of symptomatic Paget's disease (osteitis deformans). | CIBACALCIN | 1986/10/31 | Novartis Pharmaceutical Corporation | ||
| 1987/1/20 | Ifosfamide | D00343 | Treatment of testicular cancer. | IFEX | In combination with certain other approved antineoplastic agents, for third line chemotherapy in the treatment of germ cell testicular cancer. | 1988/12/30 | Bristol-Myers Squibb Pharmaceutical Research Institute |
| 1987/1/9 | Teriparatide | D06546 | Diagnostic agent to assist in establishing the diagnosis in patients presenting with clinical and laboratory evidence of hypocalcemia due to either hypoparathyroidism or pseudohypoparathyroidism. | PARATHAR | 1987/12/23 | Rhone-Poulenc Rorer Pharmaceuticals, Inc. | |
| 1986/12/8 | Leucovorin | D01211 | For use in combination with 5-fluorouracil for the treatment of metastatic colorectal cancer. | LEUCOVORIN CALCIUM | For use in combination with 5-fluorouracil to prolong survival in the palliative treatment of patients with advanced colorectal cancer. | 1991/12/12 | Immunex Corporation |
| 1986/8/20 | Botulinum toxin type A | D00783 | Treatment of cervical dystonia. | BOTOX | Treatment of cervical dystonia in adults to decrease the severity of abnormal head position and neck pain associated with cervical dystonia. | 2000/12/21 | Allergan, Inc. |
| 1986/7/30 | Erwinia L-asparaginase | D02997 | Treatment of acute lymphocytic leukemia. | ERWINASE | Treatment of patients with acute lymphoblastic leukemia (ALL) who have developed hypersensitivity to E. coli-derived asparaginase. | 2011/11/18 | Jazz Pharmaceuticals, Inc. |
| 1986/6/12 | Somatropin for injection | D02691 | For the long-term treatment of children who have growth failure due to inadequate secretion of normal endogenous growth hormone. | HUMATROPE | 1987/3/8 | Eli Lilly and Company | |
| 1986/5/15 | Trimetrexate glucuronate | D06239 | Treatment of Pneumocystis carinii pneumonia in AIDS patients. | NEUTREXIN | Concurrent leucovorin administration as an alternative therapy for the treatment of moderate to severe PCP in immunocompromised patients who are intolerant of, or are refractory to, trimethoprim sulfamethoxazole. | 1993/12/17 | Medimmune Oncology, Inc. |
| 1986/4/23 | Eflornithine HCl | D00829 | Treatment of Trypanosoma brucei gambiense infection (sleeping sickness). | ORNIDYL | 1990/11/28 | Hoechst Marion Roussel | |
| 1986/4/10 | Epoetin alfa | D03231 | Treatment of anemia associated with end stage renal disease. | EPOGEN | 1989/6/1 | Amgen, Inc. | |
| 1986/3/21 | Etidronate disodium | D00314 | Treatment of hypercalcemia of malignancy inadequately managed by dietary modification and/or oral hydration. | DIDRONEL | 1987/4/21 | MGI Pharma, Inc. | |
| 1986/2/5 | Beractant | D03096 | Treatment of neonatal respiratory distress syndrome | SURVANTA | Prevention of RDS (hyaline membrane disease) in premature infants less than 1250 grams birthweight or with evidence of surfactant deficiency | 1991/7/2 | Ross Laboratories |
| 1986/2/5 | Beractant | D03096 | Treatment of neonatal respiratory distress syndrome | SURVANTA | Treatment of ("rescue") of premature infants with RDS confirmed by x-ray and requiring mechanical ventilation | 1991/7/01 | Ross Laboratories |
| 1986/1/21 | Benzoate and phenylacetate | D10205 | For adjunctive therapy in the prevention and treatment of hyperammonemia in patients with urea cycle enzymopathy due to carbamylphosphate synthetase, ornithine, transcarbamylase, or argininosuccinate synthetase deficiency. | UCEPHAN | 1987/12/23 | ImmunexImmunex | |
| 1986/1/17 | Tiopronin | D01430 | Prevention of cystine nephrolithiasis in patients with homozygous cystinuria. | THIOLA | 1988/8/11 | Pak, Charles Y.C. M.D. | |
| 1985/12/9 | Rifampin | D00211 | For antituberculosis treatment where use of the oral form of the drug is not feasible. | RIFADIN I.V. | 1989/5/25 | Hoechst Marion Roussel | |
| 1985/12/9 | Somatrem for injection | D05884 | 1. Long-term treatment of children who have growth failure due to a lack of adequate endogenous growth hormone secretion (prevalence 15,000) 2. Treatment of short stature associated with Turner's syndrome (prevalence 8000). | PROTROPIN | 1985/10/17 | Genentech, Inc. | |
| 1985/11/14 | Mesna | D01459 | For use as a prophylactic agent in reducing the incidence of ifosfamide-induced hemorrhagic cystitis. | MESNEX | 1988/12/30 | Degussa Corporation | |
| 1985/11/6 | Zinc acetate | D06408 | Treatment of Wilson's disease. | GALZIN | For maintenance treatment of patients with Wilson's disease who have been initially treated with a chelating agent. | 1997/1/28 | Lemmon Company |
| 1985/10/21 | Methotrexate sodium | D02115 | Treatment of osteogenic sarcoma. | METHOTREXATE | For the use of high dose methotrexate with leucovorin rescue in patients with non-metastatic osteosarcoma who have undergone surgical resection or amputation for the primary tumor. | 1988/4/7 | Lederle Laboratories |
| 1985/9/25 | Epoprostenol | D0137 | Treatment of primary pulmonary hypertension. | FLOLAN | Long-term intravenous treatment of primary pulmonary hypertension in NYHA Class III and Class IV patients. | 1995/9/20 | Glaxo Wellcome Inc. |
| 1985/9/12 | Rifampin, isoniazid, pyrazinamide | D10210 | For the short-course treatment of tuberculosis. | RIFATER | Short-course treatment of tuberculosis. | 1994/5/31 | Hoechst Marion Roussel |
| 1985/8/28 | Pentastarch | D06454 | As an adjunct in leukapheresis to improve the harvesting and increase the yield of leukocytes by centrifugal means. | PENTASPAN | 1987/5/19 | DuPont Pharmaceuticals | |
| 1985/8/7 | Pentosan polysulfate sodium | D05428 | Treatment of interstitial cystitis. | ELMIRON | Relief of bladder pain or discomfort associated with interstitial cystitis. | 1996/9/26 | Alza Corporation |
| 1985/7/24 | Cromolyn sodium 4% ophthalmic solution | D00526 | Treatment of vernal keratoconjunctivitis. | OPTICROM | 1984/10/3 | Fisons Corporation | |
| 1985/7/17 | Zidovudine | D00413 | Treatment of AIDS | RETROVIR | Management of certain adult patients with symptomatic HIV infection (AIDS and advanced ARC) who have a history of cytologically confirmed Pneumocytis carinii pneumonia (PCP) or an absolute CD4 (T4 helper/induccer) lymphocyte count of less than 200/mm in the peripheral blood before therapy is begun | 1987/3/19 | Glaxo Wellcome Inc. |
| 1985/6/21 | Midodrine HCl | D01307 | Treatment of patients with symptomatic orthostatic hypotension. | AMATINE | Treatment of symptomatic orthostatic hypotension. | 1996/9/6 | Shire |
| 1985/6/7 | Calfactant | D03317 | Treatment and prevention of respiratory failure due to pulmonary surfactant deficiency in preterm infants. | INFASURF | Treatment and prevention of respiratory failure due to pulmonary surfactant deficiency in preterm infants. | 1998/7/1 | ONY, Inc. |
| 1985/3/11 | Alglucerase injection | D02810 | For replacement therapy in patients with Gaucher's disease type I. | CEREDASE | Long-term enzyme replacement therapy for children, adolescents and adult patients with a confirmed diagnosis of Type I Gaucher disease who exhibit signs and symptoms that are severe enough to result in one or more of the following conditions: a) moderate-to-severe anemia; b) thrombocytopenia with bleeding tendency; c) bone disease; d) significant hepatomegaly or splenomegaly. | 1991/4/5 | Genzyme Corporation |
| 1985/3/11 | Naltrexone HCl | D02095 | For blockade of the pharmacological effects of exogenously administered opioids as an adjunct to the maintenance of the opioid-free state in detoxified formerly opioid-dependent individuals. | TREXAN | 1984/11/30 | DuPont Pharmaceuticals | |
| 1985/2/8 | Antithrombin III (human) | D06571 | For the treatment of patients with hereditary antithrombin III deficiency in connection with surgical or obstetrical procedures or when they suffer from thromboembolism. | ATNATIV | 1989/12/13 | Pharmacia & Upjohn AB | |
| 1985/1/24 | Levomethadyl acetate hydrochloride | D00840 | Treatment of heroin addicts suitable for maintenance on opiate agonists. | ORLAAM | For the management of opiate dependence. | 1993/7/9 | Biodevelopment Corporation |
| 1985/1/16 | Factor XIII concentrate (human) | Treatment of congenital factor XIII deficiency | CORIFACT | Peri-operative management of surgical bleeding in adult and pediatric patients with congenital Factor XIII deficiency. | 2013/1/24 | CSL Behring LLC | |
| 1985/1/16 | Factor XIII concentrate (human) | Treatment of congenital factor XIII deficiency | CORIFACT | For the routine prophylactic treatment of congenital factor XIII deficiency | 2011/2/17 | CSL Behring LLC | |
| 1984/12/24 | Trientine HCl | D00736 | Treatment of patients with Wilson's disease who are intolerant, or inadequately responsive to penicillamine. | SYPRINE | 1985/11/8 | Merck Sharp & Dohme Research | |
| 1984/12/7 | Alpha1-proteinase inhibitor (human) | For replacement therapy in the alpha-1-proteinase inhibitor congenital deficiency state. | PROLASTIN | For chronic replacement therapy of individuals having congenital deficiency of alpha1- proteinase inhibitor with clinically demonstrable panacinar emphysema. | 1987/12/2 | Bayer Corporation | |
| 1984/11/26 | Antithrombin III (human) | D06571 | For replacement therapy in congenital deficiency of AT-III for prevention and treatment of thrombosis and pulmonary emboli. | THROMBATE III | 1991/12/30 | Bayer Corporation | |
| 1984/11/7 | Selegiline HCl | D00785 | As an adjuvant to levodopa and carbidopa treatment of idiopathic Parkinson's disease (paralysis agitans), postencephalitic Parkinsonism, and symptomatic Parkinsonism. | ELDEPRYL | 1989/6/5 | Somerset Pharmaceuticals, Inc. | |
| 1984/11/1 | Digoxin immune FAB (Ovine) | Treatment of potentially life threatening digitalis intoxication in patients who are refractory to management by conventional therapy. | DIGILBIND | 1986/4/22 | Glaxo Wellcome Inc. | ||
| 1984/11/1 | Potassium citrate | D05578 | Prevention of uric acid nephrolithiasis. | UROCIT-K | 1985/8/30 | University of Texas Health Science Center at Dallas | |
| 1984/11/1 | Potassium citrate | D05578 | 1. Prevention of calcium renal stones in patients with hypocitraturia 2. For avoidance of the complication of calcium stone formation in patients with uric lithiasis. | UROCIT-K | Prevention of recurring calcium conating stines associated with a deficiency in the urine. | 1985/8/7 | University of Texas Health Science Center at Dallas |
| 1984/11/1 | Teniposide | D02698 | Treatment of refractory childhood acute lymphocytic leukemia. | VUMON | Induction therapy in patients with refractory childhood acute lymphoblastic leukemia when used in combination with other approved anticancer agents. | 1992/7/14 | Bristol-Myers Squibb Pharmaceutical Research Institute |
| 1984/9/21 | Chenodiol | D00163 | For patients with radiolucent stones in well opacifying gallbladders, in whom elective surgery would be undertaken except for the presence of increased surgical risk due to systemic disease or age | CHENIX | 1983/7/28 | Solvay | |
| 1984/7/26 | Levocarnitine | D02176 | Treatment of primary and secondary carnitine deficiency of genetic origin. | CARINITOR | Treatment of primary systemic carnitine deficiency. (approved 12/27/85) Treatment of secondary carnitine deficiency. (approved 12/16/92) | 1992/12/16 | Sigma-Tau Pharmaceuticals, Inc. |
| 1984/6/11 | Clofazimine | D00278 | Treatment of lepromatous leprosy, including dapsone-resistant lepromatous leprosy and lepromatous leprosy complicated by erythema nodosum leprosum. | LAMPRENE | 1986/12/15 | Novartis Pharmaceutical Corporation | |
| 1984/5/30 | Monooctanoin | For dissolution of cholesterol gallstones retained in the common bile duct. | MOCTANIN | 1985/10/31 | Ethitek Pharmaceuticals, Inc. | ||
| 1984/5/29 | Pegademase bovine | D05384 | For enzyme replacement therapy for ADA deficiency in patients with severe combined immunodeficiency. | ADAGEN | 1990/3/21 | Sigma-tau Pharmaceuticals, Inc. | |
| 1984/5/9 | Succimer | D00572 | Treatment of lead poisoning in children. | CHEMET | 1991/1/30 | Bock Pharmacal Company | |
| 1984/3/22 | Botulinum toxin type A | D00783 | Treatment of strabismus and blepharospasms | BOTOX | Treatment of blepharospasm associated with dystonia in adults (patients 12 years of age and above) | 1989/12/30 | Allergan, Inc. |
| 1984/3/22 | Botulinum toxin type A | D00783 | Treatment of strabismus and blepharospasms | BOTOX | Treatment of strabismus associated with dystonia in adults (patients 12 years of age and above) | 1989/12/29 | Allergan, Inc. |
| 1984/3/22 | Ethanolamine oleate | D02276 | Treatment of patients with esophageal varices that have recently bled, to prevent rebleeding. | ETHAMOLIN | 1988/12/22 | QOL Medical | |
| 1984/3/16 | Hemin | D10003 | Amelioration of recurrent attacks of acute intermittent porphyria (AIP) temporarily related to the menstrual cycle in susceptible women and similar symptoms which occur in other patients with AIP, porphyria variegata and hereditary coproporphyria. | PANHEMATIN | 1983/7/20 | Abbott Laboratories | |
| 1984/3/8 | Cromolyn sodium | D00526 | Treatment of mastocytosis. | GASTROCROM | 1989/12/22 | Fisons Corporation | |
| 1984/2/28 | Levocarnitine | D02176 | Treatment of genetic carnitine deficiency. | CARNITOR | 1986/4/10 | Sigma-Tau Pharmaceuticals, Inc. | |
| 1984/2/28 | Pentamidine isethionate | D00834 | Treatment of Pneumocystis carinii pneumonia. | PENTAM 300 | 1984/10/16 | Fujisawa USA, Inc. | |
| 1984/2/9 | Altretamine | D02841 | Treatment of advanced adenocarcinoma of the ovary. | HEXALEN | Single agent palliative treatment of patients with persistent or recurrent ovarian cancer following first-line therapy with a cisplatin and/or alkylating agent based combination. | 1990/12/26 | Medimmune Oncology, Inc. |
See also: FDA Office of Orphan Products Development