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
1-(2-C-cyano-2-deoxy-B-D-arabino-pentafuranosyl)-N4-palmitoylcytosine Acute myeloid leukemia Cyclacel Limited;1 James Linday Place, Dundee DD1 5JJ; June 24, 2010
Angiotensin 1-7 To accelerate engraftment of hematopoietic cells (treatment of neutropenia, thrombocytopenia, lymphoma, and aneima)in hematopoietic stem cell transplants Tarix Pharmaceuticals, Ltd.;12 Bow Street;Cambridge, Massachusetts, 02138 June 17, 2010
Dasiprotimut-T Mantle cell lymphoma Biovest International, Inc.;324 S. Hyde Park Ave.;Tampa, Florida, 33606 June 17, 2010
N-{2-Chloro-4-[(6,7-dimethoxy-4-quinolyl)oxy]phenyl}-N’-(5-methyl-3-isoxazolyl) urea hydrochloride monohydrate Renal cancer EUSA Pharma (UK) Limited June 9, 2010
Bosentan Systemic scleroderma Actelion Ltd.;Gewerbestrasse 16, CH-4123; June 7, 2010
Anti-TCR murine monoclonal antibody (MAb, type IgM) Patients 16 years of age and younger with immune-mediated T1DM and preserved pancreatic beta cell function Tolera Therapeutics, Inc.;350 E. Michigan Avenue;Kalamazoo, Michigan, 49001 June 7, 2010
Dendritic cells pulsed with synthetic peptides derived from antigens MAGE1, HER-2, AIM-2,TRP-2, gp100 and interleukin-13 receptor alpha Glioblastoma ImmunoCellular Therapeutics Ltd.;21900 Burbank Blvd;Woodland Hills, California, 91367 June 7, 2010
Anti-thymocyte globulin [rabbit] Prophylaxis of acute organ rejection in patients receiving renal transplants Genzyme Corporation;500 Kendall Street;Cambridge, Massachusetts, 02142 May 25, 2010
N-(6-2-aminophenylamino)-6-oxohexyl)-4-methylbenzamide Friedreich Ataxia Repligen Corporation;Building 1, Suite 100;Waltham, Massachusetts, 02453 May 17, 2010
BF2.649 (Pitolisant) Narcolepsy Bioprojet Pharma;9, rue Rameau;Paris May 17, 2010
Budesonide To slow the progression of immunoglobulin A nephropathy & delay kidney failure in patients affected by the disease Calliditas Therapeutics AB;Wallingata 26b;Stockholm May 17, 2010
Bone marrow-derived mononuclear cells Buerger’s disease t2cure GmbH;Kennedyallee 93, 60596 Frankfurt am Main; May 17, 2010
Hydroxy-Propyl-Beta-Cyclodextrin Niemann Pick Disease, Type C Sphingo Biotech, LLC;14120 NW 126th Terrace;Alachua, Florida, 32615 May 17, 2010
Lentiviral vector containing the human MY07A gene Retinitis pigmentosa Sanofi US Services, Inc.;55 Corporate Drive, Mail Stop 55D-220B;Bridgewater, New Jersey, 08807 May 17, 2010
Glyeraldehyde-3-phosphate dehydrogenase Pediatric multiple sclerosis BPT Pharmaceuticals, Inc.;743 North Sweetzer Avenue #103;Los Angeles, California, 90069 April 30, 2010
Replication-incompetent GM-CSF-expressing gene-modified allogeneic chronic myeloid leukemia cancer cell lines Chronic myeloid leukemia Aduro BioTech, Inc.;740 Heinz Avenue;Berkeley, California, 94710 April 30, 2010
Auranofin Amebiasis Ctr for Discovery & Innovation in Parasitic Diseas;U. of CA, San Francisco, QB3, Byers Hall 508B;San Francisco, California, 94158 April 30, 2010
AEZS-108 (LHRH-agonist linked to doxorubicin) Ovarian cancer Aeterna Zentaris;100 Marketplace, Suite 203, 25 Mountainview Blvd;Basking Ridge, New Jersey, 07920 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
Midostaurin Mastocytosis Novartis Oncology;One Health Plaza;East Hanover, New Jersey, 07936 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
Cintredekin Besudotox Idiopathic pulmonary fibrosis Insys Development Company, Inc.;1333 South Sprectum Blvd, Suite 100;Chandler, Arizona, 85286 April 30, 2010
3,4 diaminopyridine and choline bitartrate Lambert-Eaton Myasthenic Syndrome MS Therapeutics Limited;Beechy House, Berkshire RG45 7AW; April 30, 2010
Human monoclonal antibody targeting CC-chemokine ligand 2 (CNTO 888) Ovarian cancer Ortho Biotech;200 Great Valley Parkway;Malvern, Pennsylvania, 19355 April 30, 2010