Treatments

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

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Country Product Indication Sponsor Date
MA09-hRPE cells Stargardt's macular dystrophy Astellas Pharma Global Development Inc.;1 Astellas Parkway;Northbrook, Illinois, 60062 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
Allogeneic ex vivo expanded umbilical cord blood cells Chronic myeloid leukemia Gamida Cell - Teva Joint Venture;PO Box 34670; February 1, 2010
Allogeneic ex vivo expanded unbilical cord blood cells Myelodysplastic syndromes Gamida Cell - Teva Joint Venture;PO Box 34670; February 1, 2010
Inhibitor of Tissue Factor Pathway Inhibitor (TFPI) Hemophilia A Baxter Healthcare Corporation, Baxter BioScience;One Baxter Way;Westlake Village, California, 91362 January 29, 2010
Alpha1 proteinase inhibitor (human) Emphysema secondary to congenital alpha1-antitrypsin deficiency Grifols Therapeutics, Inc.;79 TW Alexander Drive;Research Triangle Park, North Carolina, 27709 January 29, 2010
17 amino acid peptide Prevention of ischemia reperfusion injury in the lung during lung tranplantation Apeptico Forschung und Entwicklung GmbH;Mariahilferstrabe 136, Top 1150;Vienna January 29, 2010
Angiotensin 1-7 Sarcoma W. Jeffrey Petty, MD;Wake Forest University Health Sciences;Winston-Salem, North Carolina, 27157 January 29, 2010
N-[(2S)-2,3-dihydroxypropyl]-3-[(2-fluoro-4-iodophenyl)amino] isonicotinamide hydrochloride Pancreatic Cancer EMD Serono, Inc.;1 Technology Place;Rockland, Massachusetts, 02370 January 29, 2010
2-(2-phenylvinyl)-4-[-methylpiperazin-l-yl)]-6-(5-methyl-2H-pyrazol-3-yl-amino)-pyrimidine L(+) tartrate salt Acute myeloid leukemia EntreMed, Inc.;Research Triangle Park Center, Suite 400;Durham, North Carolina, 27703 January 29, 2010
(1S, 2S, 3R, 4R)-3-(5-Fluoro-2-(3-methyl-4-(4-methylpiperazin-1-yl)-phenylamino)-pyrimidin-4-ylamino)bicycle[2.2.1]hept-5-ene-2-carboxamide benzoate Acute myeloid leukemia EMD Serono, Inc.;1 Technology Place;Rockland, Massachusetts, 02370 January 19, 2010
Amitriptyline and ketamine Postherpetic neuralgia Immune Pharmaceuicals, Inc.;Alexandria Center for LIfe Sciences, 430 E. 29th St.;New York, New York, 10016 January 19, 2010
Cis-adamantane-2-spiro-3′-8′-[4′-[2′-(4′-morpholinyl)ethoxy]phenyl]-1′,2,’4′-trioxaspiro[4.5] decane mesylate Malaria Medicines for Malaria Venture (MMV);International Centre Cointrin, P. O. Box 1826;CH-1215 Geneva 15 January 19, 2010
2-[[3-({4-[(5-{2-[(3-Fluorophenyl)amino]-2-oxoethyl}-1H-pyrazol-3-yl)amino]-quinazolin-7-yl}propyl](ethyl)amino]ethyl dihydrogen phosphate trihydrate Acute myeloid leukemia AstraZeneca Pharmaceuticals LP;1800 Concord Pike, P. O. Box 8355;Wilmington, Delaware, 19803 January 19, 2010
L-asparaginase encapsulated in red blood cells Acute lymphoblastic leukemia Erytech Pharma;Batiment Adenine, 60, Avenue Rockfeller;LYON January 6, 2010
Ambrisentan Idiopathic pulmonary fibrosis Gilead Sciences, Inc.;199 East Blaine Street;Seattle, Washington, 98102 December 23, 2009
Brilliant blue G For use in ophthalmic surgery to aid in the removal of the inner limiting membrane of the eye Fluoron GmbH;Biberkopfweg 1; December 16, 2009
Burosumab X-linked hypophosphatemia (formerly known as vitamin D-resistant rickets) Ultragenyx Pharmaceutical, Inc.;60 Leveroni Court, Suite 200;Novato, California, 94949 December 14, 2009
Afamelanotide Solar urticaria Clinuvel, Inc.;P. O. Box 2176;Blue Bell, Pennsylvania, 19422 December 11, 2009
6-mercaptopurine oral liquid Acute lymphoblastic leukemia Orbona Pharma Ltd;Old Chambers, Farnham, England;Surrey December 7, 2009
Complement factor H Atypical hemolytic uremic syndrome LFB Biotechnologies S.A.;3, avenue des Tropiques, 91943 Courtaboeuf Cedex; December 7, 2009
Davunetide Progressive supranuclear palsy Allon Therapeutics, Inc.;506-1168 Hamilton Street, Vancouver, BC V6B 2S2; December 7, 2009
5′-O-(trans-9″-octadecenoyl)-1-beta-D-2’deoxy-2′,2′-difluorocytidine Pancreatic Cancer Clovis Oncology, Inc.;5500 Flatiron Parkway, Suite 100;Boulder, Colorado, 80301 December 7, 2009
Inebilizumab Scleroderma MedImmune;One MedImmune Way;Gaithersburg, Maryland, 20878 December 3, 2009
Bevacizumab In combination with a platinum and 5-FU or capecitabine for the treatment of stomach cancer Genentech, Inc.;1 DNA Way;South San Francisco, California, 94080 November 20, 2009