Category «orphan drug»

CPP 115

It’s only fair to share… (+)-(1S,4S)-4-Amino-3-(difluoromethylene)-1-cyclopentanecarboxylic acid 640897-20-7 CAS PHASE 1 NORTHWESTERN UNIVERSITY .INNOVATORS Sponsor: Catalyst Pharmaceuticals, Inc. CPP-115 free base; UNII-5TD9324Z2U; CHEMBL146927; 640897-20-7; (1S,3S)-3-Amino-4-difluoromethylenyl-1-cyclopentanoic acid; (+)-(1S,4S)-4-Amino-3-(difluoromethylene)-1-cyclopentanecarboxylic acid Molecular Formula: C7H9F2NO2 Molecular Weight: 177.151 g/mol Originator Northwestern University Developer Catalyst Pharmaceutical Partners Class Aminobutyric acids; Antiepileptic drugs; Small molecules Mechanism of Action 4-aminobutyrate transaminase inhibitors Orphan Drug Status Yes – …

Filgotinib

It’s only fair to share… Filgotinib C21H23N5O3S MW425.504 Elemental Analysis: C, 59.28; H, 5.45; N, 16.46; O, 11.28; S, 7.54 1206161-97-8 Cyclopropanecarboxamide, N-[5-[4-[(1,1-dioxido-4-thiomorpholinyl)methyl]phenyl][1,2,4]triazolo[1,5-a]pyridin-2-yl]- G146034 GLPG0634 N-(5-(4-((1,1-dioxidothiomorpholino)methyl)phenyl)-[1,2,4]triazolo[1,5-a]pyridin-2-yl)cyclopropanecarboxamide Galapagos Nv INNOVATOR PHASE 3,  Crohn’s disease, Rheumatoid arthritis, Ulcerative colitis Filgotinib is an orally available inhibitor of JAK1/JAK2 and TYK2 in phase III clinical development at Galapagos and Gilead for the …

Uridine triacetate, ウリジントリアセタート

It’s only fair to share…     Uridine triacetate Uridine, 5-hydroxy-, 2′,3′,5′-triacetate 2′,3′,5′-Tri-O-acétyluridine 223-881-5 [EINECS] CAS 4105-38-8 Priority review drug  Orphan drug FAST TRACK MF C15H18N2O9, MW 370.314 [(2R,3R,4R,5R)-3,4-bis(acetyloxy)-5-(2,4-dioxo-1,2,3,4-tetrahydropyrimidin-1-yl)oxolan-2-yl]methyl acetate Vistogard [Trade name] Xuriden [Trade name] (2R,3R,4R,5R)-2-(acetoxymethyl)-5-(2,4-dioxo-3,4-dihydropyrimidin-1(2H)-yl)tetrahydrofuran-3,4-diyl diacetate Wellstat (Originator) PN-401; RG-2133; TAU MOA:Pyrimidine analog Indication:Hereditary orotic aciduria; Chemotherapy induced poisoning To treat patients with hereditary orotic aciduria Drug …

FDA grants accelerated approval to first drug for Duchenne muscular dystrophy

It’s only fair to share… CAS 1173755-55-9 eteplirsen, eteplirsén [Spanish], étéplirsen [French] , eteplirsenum [Latin], этеплирсен [Russian], إيتيبليرسان [Arabic] Structure credit http://lgmpharma.com/eteplirsen-still-proves-efficacious-duchenne-drug/ FDA grants accelerated approval to first drug for Duchenne muscular dystrophy New therapy addresses unmet medical need The U.S. Food and Drug Administration today approved Exondys 51 (eteplirsen) injection, the first drug approved to treat patients with Duchenne muscular dystrophy …

Pitolisant

It’s only fair to share…   Pitolisant CAS 362665-56-3 FDA APPROVED 2019 AUG 1-(3-(3-(4-Chlorophenyl)propoxy)propyl)piperidine MF  C17H26ClNO MW  295.1703 HBS-101 Pitolisant Tiprolisant UNII-4BC83L4PIY   (Wakix®)Approved EU 31/3/2016, Narcolepsy A histamine H3 receptor antagonist/inverse agonist used to treat narcolepsy. BF-2649; BF-2.649; FUB-649, Ciproxidine, Tiprolisant CAS 362665-56-3, 362665-57-4 (oxalate)  CAS 903576-44-3(Pitolisant Hydrochloride) APPROVED IN EU,  European Medicine Agency (EMA) on Mar 31, 2016. BF 2.649 BF …