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
Midostaurin Mastocytosis Novartis Oncology;One Health Plaza;East Hanover, New Jersey, 07936 April 30, 2010
Midostaurin Mastocytosis Novartis Oncology;One Health Plaza;East Hanover, New Jersey, 07936 April 30, 2010
Ex-vivo cultered adult human mesenchymal stem cells Type 1 diabetes patients with residual beta cell function Mesoblast, Inc.;505 Fifth Avenue, Third Floor;New York, New York, 10017 April 30, 2010
Glyeraldehyde-3-phosphate dehydrogenase Pediatric multiple sclerosis BPT Pharmaceuticals, Inc.;743 North Sweetzer Avenue #103;Los Angeles, California, 90069 April 30, 2010
Cinacalcet 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 Amgen, Inc.;One Amgen Center Drive, Bldg 17, 1A-4-4, Mail Stop 17-1-B;Thousand Oaks, California, 91320 April 30, 2010
Fenretinide Cystic fibrosis McGill University;McGill University, Montreal, Quebec, H3A 2T5; April 7, 2010
L-ornithine phenylacetate Hepatic encephalopathy Ocera Therapeutics, Inc.;5001 South Miami Blvd, Suite 300;Durham, North Carolina, 27703 April 7, 2010
N-Acetylmannosamine (ManNAc) GNE Myopathy (Hereditary Inclusion Body Myopathy Type 2) National Human Genome Research Institute,NIH;10 Center Drive, Bldg. 10, Room 10C-103;Bethesda, Maryland, 20892 April 5, 2010
N-Acetylmannosamine (ManNAc) Hereditary inclusion body myopathy type 2 Altamira Bio, Inc. (ABIO);3 Columbus Circle, 15th Floor;New York, NY, 10019 April 5, 2010
Reversal agent linked to chloroquine-like moiety Malaria DesignMedix;2828 SW Corbett Avenue;Portland, Oregon, 97201 March 26, 2010
GNE plasmid(H001) Hereditary inclusion body myopathy type 2 HIBM Research Group;18341 Sherman Way #201A;Reseda, California, 91335 March 26, 2010
Water-miscible vitamin A palmitate Bronchopulmonary dysplasia Fox Pharma, Inc.;6097 Hidden Valley Drive;Doylestown, Pennsylvania, 18902 March 26, 2010
Anti-T-lymphocyte immune globulin, rabbit Graft Versus Host Disease Neovii Biotech NA, Inc.;430 Bedford Street, Suite 195;Lexington, Massachusetts, 02420 March 26, 2010
Fully human anti-interferon gamma monoclonal antibody Hemophagocytic lymphohistiocytosis NovImmune S.A.;14 chemin des Aulx; March 26, 2010
Chenodiol Cerebrotendinous xanthomatosis Retrophin, Inc.;3721 Valley Centre Drive, Suite 200;San Diego, California, 92130 March 22, 2010
Recombinant human glutamic acid decarboxylase 65KDa isoform Type I diabetes with residual beta cell function Diamyd Therapeutics AB;Linnegatan 89B; March 22, 2010
Recombinant Human soluble Fc-gamma Receptor IIb Idiopathic thrombocytopenic purpura Baxalta US Inc., now part of Shire Plc;650 Kendall Street;Cambridge, Massachusetts, 02142 March 22, 2010
Tirasemtiv Amyotrophic lateral sclerosis Cytokinetics Inc.;280 East Grand Avenue;South San Francisco, California, 94080 March 2, 2010
Fast skeletal muscle troponin activator Amyotrophic lateral sclerosis Cytokinetics Inc.;280 East Grand Avenue;South San Francisco, CA, 94080 March 2, 2010
Replication-incompetent GM-CSF-expressing gene-modified allogeneic acute myeloid leukemia cancer cell lines Acute myeloid leukemia Aduro BioTech, Inc.;740 Heinz Avenue;Berkeley, California, 94710 March 2, 2010
3-(6-(1-(2,2-difluorobenzo[d] [1,3]dioxyl-5-yl)cyclopropanecarboxamido)-3-methylpyridin-2-yl)benzoic acid Cystic fibrosis Vertex Pharmaceuticals Inc.;130 Waverly Street;Cambridge, Massachusetts, 02139 March 2, 2010
Replication-incompetent GM-CSF-expressing gene-modified allogeneic pancreatic cancer cell lines Pancreatic cancer Aduro BioTech, Inc.;740 Heinz Avenue;Berkeley, California, 94710 March 1, 2010
MA09-hRPE cells Stargardt's macular dystrophy Advanced Cell Technology, Inc.;33 Locke Drive, First Floor;Worcester, MA, 01605 February 2, 2010
RNA, [P-deoxy-P-(dimethylamino)] (2′,3′-dideoxy-2′,3′-imino-2′,3′-seco) (2’a?5′) (C-m5U-m5U-A-C-A-G-G-C-m5U-C-C-A-A-m5U-A-G-m5U-G-G-m5U-C-A-G-m5U), 5′ [P-[4-[[2-[2-(2-hydroxyethoxy)ethoxy]ethoxy]carbonyl]-1-piperazinyl]-N,N-dimethylaminophosphonamidate], 3′[2’a-[N2-acetyl-L-arginyl-6-aminohexanoyl-L-arginyl-L-arginyl-ß-alanyl-L-arginyl-L-arginyl-6-aminohexanoyl-L-arginyl-L-arginyl-ß-alanyl-L-arginyl-6-aminohexanoyl-ß-alanyl], octahydrochloride Duchenne muscular dystrophy AVI BioPharma International Ltd February 2, 2010
Ciprofloxacin DPI Cystic fibrosis Bayer HealthCare Pharmaceuticals, Inc.;100 Bayer Blvd, P. O. Box 915;Whippany, New Jersey, 07981 February 2, 2010