Tag «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 – …

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 …

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 …