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U.S. Department of Health and Human Services

Search Orphan Drug Designations and Approvals

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Total Results: 3500 (140 pages)     Go to page: 


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Row Num Generic Name Designation Date Orphan Designation
 1 Detailed Record for Product   ascorbic acid  05/11/2009  Treatment of Charcot-Marie-Tooth disease type 1A.
 2 Detailed Record for Product   budesonide  12/20/2006  Treatment of patients with eosinophilic esophagitis
 3 Detailed Record for Product   single chain urokinase plasminogen activator  09/11/2014  Treatment of empyema (pleural)
 4 Detailed Record for Product  (+/-)-7-[3-(4-acetyl-3-methoxy-2-propylphenoxy)propoxy]-3,4-dihydro-8-propyl-2H-1-benzopyran-2-carboxylic acid  03/31/2003  Prevention of serious adverse events associated with vascular leak syndrome caused by Interleukin-2 therapy
 5 Detailed Record for Product  (+/-)-cis-3-(4-hydroxyphenyl)-4-(4-methoxyphenyl)-3,4-dihydro-2H-cromen-7-ol  01/10/2008  Treatment of cholangiocarcinoma
 6 Detailed Record for Product  (+/-)-cis-3-(4-hydroxyphenyl)-4-(4-methoxyphenyl)-3,4-dihydro-2H-cromen-7-ol  01/10/2008  Treatment of pancreatic cancer
 7 Detailed Record for Product  (+/-)-cis-3-(4-hydroxyphenyl)-4-(4-methoxyphenyl)-3,4-dihydro-2H-cromen-7-ol  02/01/2008  Treatment of Stage IIB through Stage IV malignant melanoma
 8 Detailed Record for Product  (-)-(3aR,4S,7aR)-4-Hydroxy-4-m-tolylethynyl-octahydro-indole-1-carboxylic acid methyl ester  10/12/2011  Treatment of Fragile X syndrome
 9 Detailed Record for Product  (1-methyl-2-nitro-1H-imidazole-5-yl)methyl N,N'-bis(2-broethyl) diamidophosphate  06/05/2013  Treatment of pancreatic cancer
 10 Detailed Record for Product  (1-methyl-2-nitro-1H-imidazole-5-yl)methyl N,N'-bis(2-bromoethyl) diamidophosphate  03/09/2012  Treatment of soft tissue sarcoma
 11 Detailed Record for Product  (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  06/23/2015  Treatment of anaplastic lymphoma kinase (ALK)-positive or ROS1-positive non-small cell lung cancer
 12 Detailed Record for Product  (1R,3R,4R,5S)-3-O-[2-O-benzoyl-3-O-(sodium(2S)-3-cyclohexyl-propanoate-  02/17/2009  Treatment of vaso-occlusive crisis in patients with sickle cell disease.
 13 Detailed Record for Product  (1S)-1-(9-deazahypoxanthin-9-yl)-1,4-dideoxy-1,4-imino-D-ribitol-hydrochloride  01/29/2004  Treatment of T-cell non-Hodgkin's lymphoma
 14 Detailed Record for Product  (1S)-1-(9-deazahypoxanthin-9-yl)-1,4-dideoxy-1,4-imino-D-ribitol-hydrochloride  08/10/2004  Treatment of chronic lymphocytic leukemia and related leukemias to include prolymphocytic leukemia, adult T-cell leukemia, and hairy cell leukemia
 15 Detailed Record for Product  (1S)-1-(9-deazahypoxanthin-9-yl)-1,4-dideoxy-1,4-imino-D-ribitol-hydrochloride  08/13/2004  Treatment of acute lymphoblastic leukemia
 16 Detailed Record for Product  (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  01/19/2010  Treatment of acute myeloid leukemia
 17 Detailed Record for Product  (1S,3S)-3-amino-4-(difluoromethylene)cyclopentanecarboxylic acid hydrochloride, (1S,3S)-3-amino-4-difluoromethylenyl-1-cyclopentanoic acid hydrochloride  09/15/2010  Treatment of infantile spasms.
 18 Detailed Record for Product  (2'R,3'S)-2'-hydroxy-N-carboxy-3'-amino-5'-methyl-hexanoic,N-tert-butyl ester, 13 ester 5B-20-epoxy-1B,2a,4a,7B,9a,10a,13a-heptahydroxy-4,10-diacetate-2-benzoate-(1"S)-7,9-acrolein acetal-11(15-1)-abeotaxane  06/23/2014  Treatment of progressive supranuclear palsy
 19 Detailed Record for Product  (2E, 4E, 6Z, 8E)-3,7-dimethyl-9-(2,6,6-trimethylcyclohex-1-en-l-yl) nona-2,4,6,8-tetraen-l-yl acetate  12/02/2010  Treatment of retinitis pigmentosa
 20 Detailed Record for Product  (2E,4E,6Z,8E)-3,7-dimethyl-9-(2,6,6-trimethylcyclohex-1-enyl)nona-2,4,6,8-tetraenyl acetate 9-cis-retinyl acetate (API)  12/02/2010  Treatment of Leber congential amaurosis (LCA) due to inherited mutations in RPE65 (encoding the protein retinal pigment epithelial protein 65) or LRAT (encoding the enzyme lecithin:retinol acyltransferase)genes.
 21 Detailed Record for Product  (2S)-2-{[(2R)-2-[({[3,3-dibutyl-7-(methylthio)-1,1-dioxido-5-phenyl-2,3,4,5-tetrahydro- 1,2,5-benzothiadiazepin-8-yl]oxy}acetyl)amino]-2-(4-hydroxyphenyl)acetyl]amino}butanoic acid  10/31/2012  Treatment of primary biliary cirrhosis
 22 Detailed Record for Product  (2S)-2-{[(2R)-2-[({[3,3-dibutyl-7-(methylthio)-1,1-dioxido-5-phenyl-2,3,4,5-tetrahydro- 1,2,5-benzothiadiazepin-8-yl]oxy}acetyl)amino]-2-(4-hydroxyphenyl)acetyl]amino}butanoic acid  10/31/2012  Treatment of progressive familial intrahepatic cholestatis
 23 Detailed Record for Product  (2Z)-2-cyano-3-3hydroxy-N-[4-(trifluoromethly)phenyl]-2-hepten-6-ynamide  01/10/2005  Prevention of acute rejection following kidney, heart, and liver transplantation
 24 Detailed Record for Product  (3,5-Bis trifluoromethyl)-N-[4-methyl-3-(4-pyridin-3yl-pyrimidin-2-yl amino) phenyl] benzamide  03/18/2011  Treatment of chronic myelogenous leukemia.
 25 Detailed Record for Product  (3,5-Bis trifluoromethyl)-N-[4-methyl-3-(4-pyridin-3yl-pyrimidin-2-yl amino) phenyl] benzamide  03/18/2011  Treatment of pancreatic cancer
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