Treatments

The following database contains products that have been granted orphan status, or have been market approval in the EU or the USA.

You can filter the content by clicking on the titles: Product, Indication, Sponsor and Date. Clicking once, will filter the column alphabetically from A – Z and a second click on the same title will filter from Z – A.

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Country Product Indication Sponsor Date
Combination of nivolumab and ipilimumab Metastatic Melanoma Bristol-Myers Squibb Company;5 Research Parkway;Wallingford, Connecticut, 06492 October 9, 2014
S3,S13-cyclo(D-tyrolsyl-L-isoleucyl-L-cysteinyl-L-valyl-1-methyl-L-tryptophyl-L-glutaminyl-L-aspartyl-L-tryptophyl-N-methyl-L-glycyl-L-alanyl-L-histidyl-L-arginyl-L-cysteinyl-N-methyl-L-isoleucinamide) Hemoglobinuria Amyndas Pharmaceuticals;N.Zerva 28;Glyfada October 9, 2014
A-dmDT390-bisFv(UCHT1) Cutaneous T-cell lymphoma Angimmune, LLC;American Red Cross Holland Laboratory, 15601 Crabbs Branch Way;Rockville, Maryland, 20855 October 9, 2014
Adeno-associated viral vector serotype 9 containing the human cardiac calsequestrin gene Polymorphic Catecholergic Ventricular Tachycardia Audentes Therapeutics, Inc.;101 Montgomery St., #2650;San Francisco, California, 94104 October 2, 2014
Inhibitor of primary and secondary KIT and PDGFR alpha kinase mutants found in GIST Gastrointestinal Stromal Tumors Deciphera Pharmaceuticals, LLC;643 Massachusetts, Suite 200;Lawrence, Kansas, 66044 October 2, 2014
Theranost 68 Ga RGD A Diagnostic for the management of Moyamoya disease (MMD) Advanced Imaging Projects, LLC (AIP);3651 FAU Blvd.;Boca Raton, Florida, 33467 October 1, 2014
PEGylated recombinant human hyaluronidase PH20 Pancreatic Cancer Halozyme Therapeutics, Inc.;11388 Sorrento Valley Road;San Diego, California, 92121 October 1, 2014
Adenovirus delta 24-RGD, an oncolytic adenovirus genetically modified for treatment of glioma Glioma DNAtrix, Inc.;3210 Merryfield Row;San Diego, California, 92121 October 1, 2014
Adeno-associated virus serotype 9 expressing the human Survival Motor Neuron gene Spinal muscular atrophy AveXis, Inc.;One Energy Square, Suite 604;Dallas, Texas, 75206 September 30, 2014
Aldoxorubicin Small cell lung cancer NantCell, Inc.;9920 Jefferson Boulevard;Culver City, California, 90232 September 24, 2014
Aldoxorubicin Glioblastoma NantCell, Inc.;9920 Jefferson Boulevard;Culver City, California, 90232 September 24, 2014
Aldoxorubicin Ovarian cancer NantCell, Inc.;9920 Jefferson Boulevard;Culver City, California, 90232 September 24, 2014
Cannabidiol Glioma Insys Development Company, Inc.;1333 S. Spectrum Blvd, Suite 100;Chandler, Arizona, 85286 September 24, 2014
68G-OPS202, small somatostatin analog labeled with 68Gallium Management of gastroenteropancreatic neuroendocrine tumors Ipsen Biosciences, Inc.;650 East Kendall Street;Cambridge, Massachusetts, 02142 September 24, 2014
MV-NB-02, its bivalent ganglioside vaccine consisting of GD2-lactone and GD3-lactone each covalently conjugated to keyhole hemocyanin Neuroblastoma MabVax Therapeutics, Inc.;11588 Sorrento Road, Suite 20;San Diego, California, 92121 September 23, 2014
4-amino-1-[(1S,4R,5S)-2-fluoro-4,5-dihydroxy-3-(hydroxymethyl) cyclopent-2-en-1-yl] pyrimidin-2-one Pancreatic cancer Rexahn Pharmaceuticals, Inc.;15245 Shady Grove Road;Rockville, Maryland, 20850 September 23, 2014
Single chain urokinase plasminogen activator Empyema (pleural) Lung Therapeutics, Inc.;P. O. Box 150183;Austin, Texas, 78715 September 11, 2014
(3S)-1-azabicyclo[2.2.2]oct-3-yl {2-[2-(4-fluorophenyl)-1,3-thiazol-4-yl]propan-2-yl}carbamate Gaucher disease Genzyme;500 Kendall Street;Cambridge, Massachusetts, 02142 September 11, 2014
Cysteamine Cystic fibrosis NovaBiotics Ltd.;Cruickshank Building, Craibstone;Aberdeen AB21 9TR September 11, 2014
Vemurafenib Non-small cell lung cancer Genentech, Inc.;1 DNA Way;South San Francisco, California, 94080 September 8, 2014
5,7-dichloro-2-dimethylaminomethyl-8-hydroxyquinoline hydrochloride Huntington disease Prana Biotechnology Limited;Level 2, 369 Royal Parade, Parkville;Victoria 3052 September 4, 2014
6-((3S,4S)-4-Methyl-1-pyrimidin-2-ylmethyl-pyrrolidin-3-yl-1-(tetrahydro-pyran-4-yl)-1,5-dihydro-pryazolo[3,4-d]pyrimidin-4-one Sickle cell anemia Pfizer, Inc.;500 Arcola Road;Collegeville, Pennsylvania, 19426 September 4, 2014
N-(2-{2-dimethylaminoethyl-methylamino}-4-methoxy-5-{[4-(1-methylindol-3-yl)pyrimidin-2-yl]amino}phenyl)prop-2-enamide mesylate salt Epidermal growth factor receptor mutation-positive non-small cell lung cancer AstraZeneca Pharmaceuticals LP;P. O. Box 8355, 1800 Concord Pike;Wilmington, DE, 19803-8355 September 4, 2014
Osimertinib Non-small cell lung cancer AstraZeneca Pharmaceuticals LP;P. O. Box 8355, 1800 Concord Pike;Wilmington, Delaware, 19803 September 4, 2014
5,7-dichloro-2-methylaminomethyl-8-hydroxyquinolone hydrochloride Huntington disease Prana Biotechnology Limited;Level 2, 369 Royal Parade, Parkville;Victoria 3052 September 4, 2014