Category «orphan drug»

Uridine triacetate

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 Hydrochloride

It’s only fair to share…   Pitolisant 1-(3-(3-(4-Chlorophenyl)propoxy)propyl)piperidine MF  C17H26ClNO MW  295.1703 (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) 1-{3-[3-(4-Chlorophenyl)propoxy]propyl}piperidine hydrochloride (1:1) Molecular Weight 332.31 Formula C17H26ClNO ● HCl Bioprojet INNOVATOR Jean-Charles Schwartz, Jeanne-Marie Lecomte Pitolisant (INN) or tiprolisant (USAN) is a histamine …