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U.S. Department of Health and Human Services

Search Orphan Drug Designations and Approvals

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Total Results: 3244 (130 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 02-01-2008 Treatment of Stage IIB through Stage IV malignant melanoma
7 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
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(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.
12 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
13 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
14 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
15 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
16 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.
17 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
18 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
19 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.
20 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
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 progressive familial intrahepatic cholestatis
22 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
23 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
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] benzamideNRC-AN-019 03-18-2011 Treatment of Glioma
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Total Results: 3244 (130 pages)     Go to page: 


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