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
Blinatumomab Acute lymphoblastic leukemia Amgen, Inc.;One Amgen Center Dr., Mail Stop 17-2-A;Thousand Oaks, California, 91320 May 16, 2008
Blinatumomab Treatment for hairy cell leukemia Amgen, Inc.;One Amgen Center Drive;Thousand Oaks, California May 16, 2008
Blinatumomab Chronic lymphocytic leukemia Amgen, Inc.;One Amgen Center Drive;Thousand Oaks, California May 16, 2008
Dimethyl sulfoxide For use in combination with antimicrobial drugs for the treatment of drug resistant tuberculosis Abela Pharmaceuticals, Inc.;21581 Midcrest Drive;Lake Forest, California, 92630 May 9, 2008
Cysteamine Huntington disease Horizon Pharma USA, Inc.;150 S. Saunders Road, Suite 100;Lake Forest, Illinois, 60045 May 9, 2008
Palifosfamide Soft tissue sarcomas ZIOPHARM Oncology, Inc.;One First Avenue, Parris Building #34, Navy Yard Plaza;Boston, Massachusetts, 02129 May 5, 2008
Ammonium tetrathiomolybdate Idiopathic pulmonary fibrosis Pipex Pharmaceuticals, Inc.;3930 Varsity Drive;Ann Arbor, Michigan, 48108 May 5, 2008
Eltrombopag Idiopathic thrombocytopenia purpura Novartis Pharmaceuticals Corp.;One Health Plaza;East Hanover, New Jersey, 07936 May 5, 2008
(R)-4-(3-morpholin-4-yl-1-phenylsulfanylmethyl-propylamino)-N-(4-{4-[2-(4-chlorophenyl)-5,5-dimethylcyclohex-1-enylmethyl]-piperazin-1-yl}-benzoyl)-3-trifluoromethanesulfonylbenzenesulfonamide bis-hydrochloride Multiple myeloma AbbVie, Inc.;1 N Waukegan Road, AP34/RA72;North Chicago, Illinois, 60064 May 5, 2008
Valproate Fragile X syndrome Neuropharm Ltd,;Fetcham Park House, Surrey KT22 9HD; May 5, 2008
Cyclosporine A Herpes simplex virus stromal keratitis Santen SAS;1 rue Pierre Fontaine, 91058 Evry cedex; April 9, 2008
Anti-von Willebrand Aptamer Thrombotic thrombocytopenic purpura Archemix Corporation;300 Third Street;Cambridge, Massachusetts, 02142 April 9, 2008
6-alpha-ethylchenodeoxycholic acid Primary sclerosing cholangitis Intercept Pharmaceuticals, Inc.;18 Desbrosses Street;New York, New York, 10013 April 9, 2008
Alpha1-antitrypsin(human) Bronchiectasis Kamada Ltd.;7 Sapir Street;Ness Ziona April 3, 2008
Autologous lymphocytes depleted ex vivo of immunoreactive T cells using 4,5 Graft Versus Host Disease Kiadis Pharma Canada, Inc.;2525 Marie Curie; April 3, 2008
Human autologous bone-forming cell derived from bone marrow stem cells Osteonecrosis Bone Therapeutics S.A.;Rue Auguste Piccard, 37;Gosselies March 24, 2008
Bifidobacterium infantis 35624 Ulcerative colitis Alimentary Health Limited;Cork Airport Business Park, Building 2800, Cork; March 24, 2008
5-[(E)-2-(4-hydroxyphenyl)-ethenyl] benzene-1,3 diol Mitochondrial encephalomyopathy lactic acidosis and stroke-like episodes Sirtris Pharmaceuticals, Inc.;200 Technology Square;Cambridge, Massachusetts, 02139 March 13, 2008
Pacritinib Myelofibrosis CTI BioPharma Corporation;3101 Western Avenue, Suite 600;Seattle, Washington, 98121 March 13, 2008
Ataluren Spinal muscular atrophy PTC Therapeutics, Inc.;100 Corporate Court;South Plainfield, New Jersey, 07080 March 10, 2008
Amrubicin Small cell lung cancer Celgene Corporation;9900 W. 109th Street, Suite 300;Overland Park, Kansas, 66210 March 10, 2008
N2′-Deacetyl-N2′-[4-methyl-4-(oxobuthyldithio)-1-oxopentyl]-maytansine-chimerized anti-CD138 IgG4 Monoclonal Antibody Multiple myeloma Biotest Pharmaceuticals Corporation;5800 Park of Commerce Blvd, NW;Boca Raton, Florida, 33487 February 27, 2008
Omegaven emulsion Parenteral nutrition-associated liver disease Fresenius Kabi USA, LLC;3 Corporate Drive;Lake Zurich, Illinois, 60047 February 27, 2008
Levofloxacin Cystic fibrosis Horizon Pharma USA, Inc.;150 S. Saunders Road, Suite 100;Lake Forest, Illinois, 60045 February 27, 2008
Arylsulfatase A (rhASA) Metachromatic leukodystrophy Shire Human Genetic Therapies, Inc.;300 Shire Way;Lexington, Massachusetts, 02421 February 27, 2008