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Orphan Drug Designation List Orphan Drug Designations and Approvals List as of 03‐03‐2014 Governs April 1, 2014 ‐ Jun 30, 2014 Row Contact Generic Name Trade Name Designation Date Designation Num Company/Sponsor (3S)‐3‐{4‐[7‐(aminocarbonyl)‐ (3s)‐3‐{4‐[7‐ 2 2H‐indazol‐2‐ (Aminocarbonyl)‐2 4/30/2010 Treatment of ovarian cancer TESARO, Inc. Treatment of Charcot‐Marie‐ 3 ascorbic acid n/a 5/11/2009 Tooth disease type 1A. Murigenetics SAS Treatment of patients with Meritage Pharma, 4 budesonide n/a 12/20/2006 eosinophilic esophagitis. Inc. (‐)‐(3aR,4S,7aR)‐4‐Hydroxy‐4‐ m‐tolylethynyl‐octahydro‐ Novartis indole‐1‐carboxylic acid Pharmaceuticals 5 methyl ester n/a 10/12/2011 Treatment of Fragile X syndrome Corp. (1‐methyl‐2‐nitro‐1H‐ imidazole‐5‐yl)methyl N,N'‐ bis(2‐broethyl) 6 diamidophosphate n/a 6/5/2013 Treatment of pancreatic cancer EMD Serono (1‐methyl‐2‐nitro‐1H‐ imidazole‐5‐yl)methyl N,N'‐ bis(2‐bromoethyl) Threshold 7 diamidophosphate n/a 3/9/2012 Treatment of soft tissue sarcoma Pharmaceuticals, Inc. (1R,3R,4R,5S)‐3‐O‐[2‐O‐ Treatment of vaso‐occlusive benzoyl‐3‐O‐(sodium(2S)‐3‐ crisis in patients with sickle cell 8 cyclohexyl‐propanoate‐ n/a 2/17/2009 disease. GlycoMimetics, Inc. (1S)‐1‐(9‐deazahypoxanthin‐9‐ yl)‐1,4‐dideoxy‐1,4‐imino‐D‐ Treatment of acute Mundipharma 9 ribitol‐hydrochloride n/a 8/13/2004 lymphoblastic leukemia Research Limited Page 1 of 273 Orphan Drug Designations and Approvals List as of 03‐03‐2014 Governs April 1, 2014 ‐ Jun 30, 2014 Row Contact Generic Name Trade Name Designation Date Designation Num Company/Sponsor Treatment of chronic lymphocytic leukemia and related leukemias to include (1S)‐1‐(9‐deazahypoxanthin‐9‐ prolymphocytic leukemia, adult T‐ yl)‐1,4‐dideoxy‐1,4‐imino‐D‐ cell leukemia, and hairy cell Mundipharma 10 ribitol‐hydrochloride n/a 8/10/2004 leukemia Research Ltd. (1S)‐1‐(9‐deazahypoxanthin‐9‐ yl)‐1,4‐dideoxy‐1,4‐imino‐D‐ Treatment of T‐cell non‐ Mundipharma 11 ribitol‐hydrochloride n/a 1/29/2004 Hodgkin's lymphoma Research Limited (1S,3S)‐3‐amino‐4‐ (difluoromethylene)cyclopenta necarboxylic acid hydrochloride, (1S,3S)‐3‐amino‐ 4‐difluoromethylenyl‐1‐ Catalyst cyclopentanoic acid Pharmaceutical 12 hydrochloride n/a 9/15/2010 Treatment of infantile spasms. Partners (2E, 4E, 6Z, 8E)‐3,7‐dimethyl‐9‐ (2,6,6‐trimethylcyclohex‐1‐en‐l‐ yl) nona‐2,4,6,8‐tetraen‐l‐yl Treatment of retinitis 13 acetate n/a 12/2/2010 pigmentosa QLT Inc. Treatment of Leber congential amaurosis (LCA) due to inherited mutations in RPE65 (encoding (2E,4E,6Z,8E)‐3,7‐dimethyl‐9‐ the protein retinal pigment (2,6,6‐trimethylcyclohex‐1‐ epithelial protein 65) or LRAT enyl)nona‐2,4,6,8‐tetraenyl (encoding the enzyme acetate 9‐cis‐retinyl acetate lecithin:retinol 14 (API) n/a 12/2/2010 acyltransferase)genes. QLT, Inc. Page 2 of 273 Orphan Drug Designations and Approvals List as of 03‐03‐2014 Governs April 1, 2014 ‐ Jun 30, 2014 Row Contact Generic Name Trade Name Designation Date Designation Num Company/Sponsor (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}b Treatment of primary biliary 15 utanoic acid n/a 10/31/2012 cirrhosis Albireo AB (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}b Treatment of progressive familial 16 utanoic acid n/a 10/31/2012 intrahepatic cholestatis Albireo AB (2Z)‐2‐cyano‐3‐3hydroxy‐N‐[4‐ Prevention of acute rejection (trifluoromethly)phenyl]‐2‐ following kidney, heart, and liver Fujisawa Healthcare, 17 hepten‐6‐ynamide Fk778 1/10/2005 transplantation Inc. (3,5‐Bis trifluoromethyl)‐N‐[4‐ methyl‐3‐(4‐pyridin‐3yl‐ pyrimidin‐2‐yl amino) phenyl] Treatment of chronic NATCO Pharma 18 benzamide n/a 3/18/2011 myelogenous leukemia. Limited (3,5‐Bis trifluoromethyl)‐N‐[4‐ methyl‐3‐(4‐pyridin‐3yl‐ pyrimidin‐2‐yl amino) phenyl] NATCO Pharma 19 benzamide n/a 3/18/2011 Treatment of pancreatic cancer Limited Page 3 of 273 Orphan Drug Designations and Approvals List as of 03‐03‐2014 Governs April 1, 2014 ‐ Jun 30, 2014 Row Contact Generic Name Trade Name Designation Date Designation Num Company/Sponsor (3,5‐Bis trifluoromethyl)‐N‐[4‐ methyl‐3‐(4‐pyridin‐3yl‐ pyrimidin‐2‐yl amino) phenyl] NATCO Pharma 20 benzamideNRC‐AN‐019 n/a 3/18/2011 Treatment of Glioma Limited (3S)‐3‐(4‐ trifluoromethoxybenzyloxy)‐6‐ nitro‐2H‐3,4‐ Global Alliance for TB 21 dihydroimidazo[2,1‐b]oxazine n/a 7/5/2007 Treatment of tuberculosis. Drug Development (3S)‐3‐[(2S)‐2‐({N‐[2‐tert‐ butyl)phenyl]carbamoyl}carbo nylamino) propanoylamino]‐4‐ oxo‐5‐(2,3,5,6‐ Treatment of patients Pfizer Global tetrafluorophenoxy) pentanoci undergoing solid organ Research and 22 acid n/a 8/19/2003 transplantation. Development (4R,5R)‐1‐[[4‐[[4‐[3,3‐Dibutyl‐7‐ (dimethylamino)‐2,3,4,5‐ tetrahydro‐4‐hydroxy‐1,1‐ dioxido‐1‐benzothiepin‐5‐ yl]phenoxy]methyl]phenyl]met hyl]‐4‐aza‐l‐ azoniabicyclo[2.2.2]octane Treatment of progressive familial Lumena 23 Chloride n/a 9/4/2013 intrahepatic cholestasis Pharmaceuticals. Inc. Page 4 of 273 Orphan Drug Designations and Approvals List as of 03‐03‐2014 Governs April 1, 2014 ‐ Jun 30, 2014 Row Contact Generic Name Trade Name Designation Date Designation Num Company/Sponsor (4R,5R)‐1‐[[4‐[[4‐[3,3‐Dibutyl‐7‐ (dimethylamino)‐2,3,4,5‐ tetrahydro‐4‐hydroxy‐1,1‐ dioxido‐1‐benzothiepin‐5‐ yl]phenoxy]methyl]phenyl]met hyl]‐4‐aza‐l‐ azoniabicyclo[2.2.2]octane Treatment of primary biliary Lumena 24 Chloride n/a 9/4/2013 cirrhosis Pharmaceuticals, Inc. (4R,5R)‐1‐[[4‐[[4‐[3,3‐Dibutyl‐7‐ (dimethylamino)‐2,3,4,5‐ tetrahydro‐4‐hydroxy‐1,1‐ dioxido‐1‐benzothiepin‐5‐ yl]phenoxy]methyl]phenyl]met hyl]‐4‐aza‐l‐ azoniabicyclo[2.2.2]octane Lumena 25 Chloride n/a 9/4/2013 Treatment of alagille syndrome Pharmaceuticals, Inc. (4R,5R)‐1‐[[4‐[[4‐[3,3‐Dibutyl‐7‐ (dimethylamino)‐2,3,4,5‐ tetrahydro‐4‐hydroxy‐1,1‐ dioxido‐1‐benzothiepin‐5‐ yl]phenoxy]methyl]phenyl]met hyl]‐4‐aza‐l‐ azoniabicyclo[2.2.2]octane Treatment of primary sclerosing Lumena 26 Chloride n/a 9/4/2013 cholangitis Pharmaceuticals' Inc. (5R)‐5‐(4‐{[2‐ fluorophenyl)methyl]oxy}phen yl)‐L‐prolinamide, Treatment of trigeminal Convergence 27 hydrochloride n/a 7/24/2013 neuralgia Pharmaceuticals Ltd. Page 5 of 273 Orphan Drug Designations and Approvals List as of 03‐03‐2014 Governs April 1, 2014 ‐ Jun 30, 2014 Row Contact Generic Name Trade Name Designation Date Designation Num Company/Sponsor (6‐[4‐Deoxy‐4‐[(2E,4E)‐ Parenteral treatment of painful, tetradecadienoylglycyl] amino‐ chronic, chemotherapy‐induced L‐glyceroB‐L‐manno‐ peripheral neuropathy that is heptopyranosyl]amino‐9H‐ refractory to conventional DARA BioSciences, 28 purine) n/a 2/21/2014 analgesics Inc. (6‐ maleimidocaproyl)hydrazone 29 of doxorubicin n/a 6/29/2011 Treatment of soft tissue sarcoma CytRx Corporation For use in combination with 5‐ (6R,S)5,10‐methylene‐ fluorouracil for the treatment of Adventrx 30 tetrahydrofolic acid Cofactor 8/13/2004 patients with pancreatic cancer Pharmaceuticals, Inc. (9‐[N‐(3‐morpholinopropyl)‐ sulfonyl]‐5,6‐dihydro‐5‐oxo‐11‐ Prevention of post‐operative Inotek H‐indeno [1,2‐c] isoquinoline complications of aortic anuerysm Pharmaceuticals 31 methanesulfonic acid n/a 12/8/2004 surgical repair Corporation (E)‐4‐carboxystyryl‐4‐ chlorobenzyl‐sulfone, sodium Treatment of acute radiation Onconova 32 salt Ex‐Rad(R) 9/4/2012 syndrome Therapeutics, Inc. (E)‐4‐carboxystyryl‐4‐ Prevention of acute radiation chlorobenzyl‐sulfone, sodium toxicity, also known as Acute Onconova 33 salt Ex‐Rad(R) 6/1/2012 Radiation Syndrome (ARS) Therapeutics, Inc. (N‐[2,6‐bis(1‐methylethyl)‐ pheyl‐N'‐[[1‐4‐dimethyl‐ amino)phenyl]cyclopentyl]met Treatment of adrenocortical 34 hyl]urea, hydrochloride salt n/a 3/9/2012 carcinoma Atterocor, Inc. Page 6 of 273 Orphan Drug Designations and Approvals List as of 03‐03‐2014 Governs April 1, 2014 ‐ Jun 30, 2014 Row Contact Generic Name Trade Name Designation Date Designation Num Company/Sponsor (R)‐1‐phenylethyl‐5‐(4‐ biphenyl‐4‐ cyclopropanecarboxylic acid)‐3‐ methylisoxazole‐4‐yl Treatment of idiopathic Bristol‐Myers Squibb 35 carbamate sodium salt n/a 4/15/2011 pulmonary fibrosis Company (R)‐2‐methyl‐6‐nitro‐2‐{4‐[4‐(4‐ trifluoromethoxyphenoxy)pipe Otsuka ridin‐1‐yl]phenoxymethyl}‐2,3‐ Treatment of pulmonary Pharmaceutical 36 dihydroimidazo[2,1‐b]oxazole n/a 7/12/2007 tuberculosis. Company, Ltd (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‐ trifluoromethanesulfonylbenze nesulfonamide bis‐ Treatment of small cell lung 37 hydrochloride n/a 12/19/2007 cancer. AbbVie, Inc Treatment of neuroleptic‐ (R)‐N‐[2‐(6‐Chloro‐methoxy‐1H‐ induced tardive dyskinesia in Phase 2 Discovery, 38 indol‐3yl)propyl]acetamide n/a 7/3/2003 schizophrenia patients Inc. (R)‐N‐[2‐(6‐chloro‐5‐methoxy‐ Treatment of circadian rhythm 1H‐indol‐3‐ sleep disorders in blind people Phase 2 Discovery, 39 yl)propyl]acetamide n/a 10/3/2001 with no light perception Inc. Page 7 of 273 Orphan Drug Designations and Approvals List as of 03‐03‐2014 Governs April 1, 2014 ‐ Jun 30, 2014 Row Contact Generic Name Trade Name Designation Date Designation Num Company/Sponsor (S)‐10‐ [(dimethylamino)methyl]‐4‐ ethyl‐9hydroxy‐4‐O‐[a‐ (2",4",5",7"‐tetranitro‐9"‐ fluorenylideneaminooxy)propi onyl]‐ 1H‐pyrano[3',4',6',7',] indolizino [1,2‐B]‐quinoline‐3, TLC 14‐(4H, 12H)‐dione, Treatment of hepatocellular Biopharmaceuticals, 40 hydrochloride Lipotecan 10/6/2010 carcinoma Inc.
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