Category «Orphan Drug Status»

VT 1129

It’s only fair to share… VT 1129 1340593-70-5 CAS MF C22 H14 F7 N5 O2, MW 513.37 2-Pyridineethanol, α-(2,4-difluorophenyl)-β,β-difluoro-α-(1H-tetrazol-1-ylmethyl)-5-[4-(trifluoromethoxy)phenyl]-, (αR)- R ISOMER ROTATION + Originator Viamet Pharmaceuticals Class Antifungals; Small molecules Mechanism of Action 14-alpha demethylase inhibitors Orphan Drug Status Yes – Cryptococcosis On Fast track Cryptococcosis Phase I Cryptococcosis Most Recent Events 01 Jun 2016 VT 1129 receives Fast Track designation for Cryptococcosis …

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 …

DROXIDOPA

It’s only fair to share… DROXIDOPA ORPHAN DRUG, CAS 23651-95-8, 3916-18-5 ROTATION   –FORM (2S,3R)-2-amino-3-(3,4-dihydroxyphenyl)-3-hydroxypropanoic acid 223-480-5 [EINECS], L-Tyrosine, β,3-dihydroxy-, (βR)-, L-Tyrosine, β,3-dihydroxy-, threo- Northera [Trade name] threo-b,3-Dihydroxy-L-tyrosine 1-14-00-00685 [Beilstein], L-threo-3-(3,4-Dihydroxyphenyl)serine, L-Dihydroxyphenylserine, L-DOPS;DOPS, \ Northera, SM5688, L-DOPS|Northera®, |SM-5688, L-threo-dihydroxyphenylserine, L-Threodops L-threo-droxidopa, threo-2-Amino-3-(3,4-dihydroxyphenyl)-3-hydroxypropionic acid THREO-DIHYDROXYPHENYLSERINE threo-Dopaserine UNII:24A0V01WKS UNII:J7A92W69L7 FDA 2/18/2014, FDA approves Northera to treat neurogenic orthostatic hypotension Properties: Crystals from ethanol and ether, mp 232-235° (dec). [a]D20 -39° …

Evofosfamide, эвофосфамид , إيفوفوسفاميد , 艾伏磷酰胺 ,

It’s only fair to share…   Evofosfamide, HAP-302 , TH-302, TH 302 эвофосфамид ,  إيفوفوسفاميد ,  艾伏磷酰胺 , Molecular Formula C9H16Br2N5O4P Average mass 449.036 Da (1-Methyl-2-nitro-1H-imidazol-5-yl)methyl N,N’-bis(2-bromoethyl)phosphorodiamidate (1-Methyl-2-nitro-1H-imidazol-5-yl)methyl-N,N’-bis(2-bromethyl)phosphorodiamidat 918633-87-1 TH-302 is a nitroimidazole-linked prodrug of a brominated derivative of an isophosphoramide mustard previously used in cancer drugs Originator Threshold Pharmaceuticals Developer Merck KGaA; Threshold Pharmaceuticals Class Antineoplastics; Nitroimidazoles; Phosphoramide mustards; Small …