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
Melphalan Bile duct cancer Delcath Systems, Inc.;1633 Broadway, Suite 22C;New York, New York, 10019 July 14, 2015
Vitamin A palmitate Bronchopulmonary dysplasia Advent Therapeutics, Inc.;6500 Old Carversville Road;Lumberville, Pennsylvania, 18933 July 14, 2015
Recombinant multimerized human IgG1 Fc Chronic inflammatory demyelinating polyneuropathy Pfizer, Inc.;500 Arcola Road;Collegeville, Pennsylvania, 19426 July 14, 2015
Adenovirus vaccine encoding reduced expression in immortalized cell protein Malignant pleural mesothelioma MTG Biotherapeutics;8195 Run of the Knolls Court;San Diego, California, 92127 July 14, 2015
Thioureidobutyronitrile Ovarian cancer Innovation Pharmaceuticals, Inc.;100 Cummings Center, Suite 151-B;Beverly, Massachusetts, 01915 July 14, 2015
Nano-diamino-tetraiodothyroacetic acid Glioblastoma NanoPharmaceuticals, LLC;1 Discovery Place;Rensselaer, New York, 12144 July 9, 2015
Propranolol and etodolac Glioma Vicus Therapeutics, LLC;55 Madison Avenue, Suite 400;Morristown, New Jersey, 07960 July 8, 2015
1-(4-(benzylamino)-7,8-dihydro-5H-pyrano[4,3-d]pyrimidin-2-yl)-2-methyl-1H-indole-4-carboxamide Multiple myeloma Cleave Biosciences, Inc.;866 Malcolm Road, Suite 100;Burlingame, California, 94010 July 8, 2015
Sarizotan Rett like syndrome Newron Pharmaceuticals US, Inc.;89 Headquarter Plaza North, Suite 1438;Morristown, New Jersey, 07960 July 7, 2015
ANA-conjugated dactunomycin nanoemulsion Ewing sarcoma NanoSmart Pharmaceuticals, Inc.;27071 Cabot Road, Suite 104;Laguna Hills, California, 92653 July 7, 2015
Gemcitabine ready-to-use Ovarian cancer Sun Pharmaceutical Industries Ltd.;Acme Plaza, Andheri (East), Mumbai 400059; June 24, 2015
Gemcitabine ready-to-use Pancreatic Cancer Sun Pharmaceutical Industries Ltd.;Acme Plaza, Andheri (East), Mumbai 400059; June 24, 2015
(1OR)-7-amino-12-fluoro-2,10,16-trimethyl-15 oxo-10,15,16,17-tetrahydro-2H-8,4-(metheno)pyrazolo[4,3-h][2,5,11]benzoxadiazacyclotetradecine-3-carbonitrile Non-small cell lung cancer Pfizer, Inc.;235 East 42nd Street (M/S 219/9/30);New York, New York, 10017 June 23, 2015
Volanesorsen sodium, apolipotrotein C-III antisense oligonucleotide Hyperlipoproteinemia type 1 Akcea Therapeutics, Inc.;55 Cambridge Parkway, Suite 100;Cambridge, Massachusetts, 02142 June 23, 2015
2-{3-[(3R)-3-({[2-chloro-3-(trifluoromethyl)phenyl]methyl}(2,2-diphenylethyl)amino)butoxy]phenyl}acetic acid Malignant melanoma stages IIB to IV Rgenix, Inc.;310 East 67th St., Suite 1-12;New York, New York, 10065 June 23, 2015
Chlorotoxin conjugated to indocyanine green dye Diagnostic for the management of malignant brain tumors Blaze Bioscience, Inc.;530 Fairview Avenue, North, Suite 1400;Seattle, Washington, 98109 June 23, 2015
Antisense oligonucleotide directed against TGF-beta2 mRNA Prevention of scarring post glaucoma filtration surgery Isarna Therapeutics GmbH June 19, 2015
6-ethoxy-7-methoxy-2-(2-methylsulfanylphenyl)-3,1-bensoxazin-4-one Netherton syndrome Sixera Pharma AB;Erik Dahlbergsgaten 11a2t, Vastra Gotaland 41126; June 18, 2015
Pembrolizumab Gastric cancer, including gastroesophageal junction adenocarcinoma Merck Research Laboratories;351 North Sumneytown Pike, UG2C-50;North Wales, Pennsylvania, 19454 June 16, 2015
2-{3-[(3R)-3-({[2-chloro-3-(trifluoromethyl)phenyl]methyl}(2,2-diphenylethyl)amino)butoxy]phenyl}acetic acid Glioblastoma Rgenix, Inc.;310 East 67th St., Suite 1-12;New York, New York, 10065 June 16, 2015
2-{3-[(3R)-3-({[2-chloro-3-(trifluoromethyl)phenyl]methyl}(2,2-diphenylethyl)amino)butoxy]phenyl}acetic acid Ovarian cancer Rgenix, Inc.;310 East 67th St., Suite 1-12;New York, New York, 10065 June 16, 2015
Synthetic signal peptide of human mucin-1 (amino acids 1-21) Multiple myeloma Vaxil Bio Therapeutics Ltd.;13 Einstein St., Building 13A, Nes-Ziona 7414001; June 16, 2015
Recombinant adeno-associated virus serotype rhesus 74 (rh74) expressing the human CT GalNac transferase (GALGT2) gene, also called the Sda or Cad GAlNac transferase or b4Galnt2, under control of the muscle creatine kinase (MCK) promoter Duchenne muscular dystrophy Nationwide Children's Hospital;700 Children's Drive, Rm WA2025;Columbus, Ohio, 43205 June 16, 2015
Imetelstat Myelofibrosis Janssen Research & Development, LLC;P. O. Box 300, 920 US Route 202;Raritan, New Jersey, 08869 June 11, 2015
(6aR, 10aR)-3-(1’,1’-dimethylheptyl)-Δ8-tetrahydro-cannabinol-9-carboxylic acid Systemic sclerosis Corbus Pharmaceuticals, Inc.;100 River Ridge Drive, Suite 103;Norwood, MA, 02062 June 10, 2015