Tag «orphan drug»

TAFAMIDIS

It’s only fair to share… Tafamidis Molecular Formula C14H7Cl2NO3 Average mass 308.116 Da TAFAMIDIS, Fx-1006A PF-06291826 2-(3,5-Dichlorophenyl)-1,3-benzoxazole-6-carboxylic acid 594839-88-0 [RN] 6-Benzoxazolecarboxylic acid, 2-(3,5-dichlorophenyl)- Vyndaqel Tafamidis meglumine Familial amyloid polyneuropathy LAUNCHED PFIZER 2011 EU ApprovedJapanese Pharmaceuticals and Medical Devices Agency in September 2013 PHASE 3, at  FDA, Amyloidosis, PFIZER Molecular Formula: C21H24Cl2N2O8 Molecular Weight: 503.329 g/mol CAS 951395-08-7 http://www.ema.europa.eu/docs/en_GB/document_library/EPAR_-_Public_assessment_report/human/002294/WC500117838.pdf D-Glucitol, 1-deoxy-1-(methylamino)-, 2-(3,5-dichlorophenyl)-6-benzoxazolecarboxylate Tafamidis (INN, or Fx-1006A,[1] trade …

BMS-986020

It’s only fair to share… BMS-986020 AM-152; BMS-986020; BMS-986202 cas 1257213-50-5 Chemical Formula: C29H26N2O5 Molecular Weight: 482.536 (R)-1-(4′-(3-methyl-4-(((1-phenylethoxy)carbonyl)amino)isoxazol-5-yl)-[1,1′-biphenyl]-4-yl)cyclopropane-1-carboxylic acid Cyclopropanecarboxylic acid, 1-(4′-(3-methyl-4-((((1R)-1-phenylethoxy)carbonyl)amino)-5-isoxazolyl)(1,1′-biphenyl)-4-yl)- 1-(4′-(3-Methyl-4-(((((R)-1-phenylethyl)oxy)carbonyl)amino)isoxazol-5-yl)biphenyl-4-yl)cyclopropanecarboxylic acid UNII: 38CTP01B4L For treatment for pulmonary fibrosis, phase 2, The lysophosphatidic acid receptor, LPA1, has been implicated as a therapeutic target for fibrotic disorders Lysophospholipids (LPs), including lysophosphatidic acid (LPA), sphingosine 1-phospate (S1P), …

FDA approves Mylotarg (gemtuzumab ozogamicin) for treatment of acute myeloid leukemia

It’s only fair to share… FDA approves Mylotarg for treatment of acute myeloid leukemia 09/01/2017 The U.S. Food and Drug Administration today approved Mylotarg (gemtuzumab ozogamicin) for the treatment of adults with newly diagnosed acute myeloid leukemia whose tumors express the CD33 antigen (CD33-positive AML). The FDA also approved Mylotarg for the treatment of patients …

FDA approves first subcutaneous C1 Esterase Inhibitor to treat rare genetic disease

It’s only fair to share… FDA approves first subcutaneous C1 Esterase Inhibitor to treat rare genetic disease 06/22/2017   The U.S. Food and Drug Administration today approved Haegarda, the first C1 Esterase Inhibitor (Human) for subcutaneous (under the skin) administration to prevent Hereditary Angioedema (HAE) attacks in adolescent and adult patients. The subcutaneous route of …

TRIENTINE HYDROCHLORIDE, 塩酸トリエンチン , 曲恩汀

It’s only fair to share… TRIENTINE Molecular Formula C6H18N4 Average mass 146.234 Da 112-24-3 CAS 曲恩汀, KD-034, MK-0681, MK-681, TECZA, TETA, TJA-250 1,2-Ethanediamine, N1,N2-bis(2-aminoethyl)- 1,8-diamino-3,6-diazaoctane TRIENTINE HYDROCHLORIDE Molecular Formula C6H19ClN4 Average mass 182.695 Da 38260-01-4 CAS Launched – 1986 VALEANT, WILSONS DISEASE 塩酸トリエンチン Trientine Hydrochloride C6H18N4▪2HCl : 219.16 [38260-01-4] Aton Pharma, a subsidiary of Valeant Pharmaceuticals, has developed and launched Syprine, a capsule formulation of trientine hydrochloride, …

Lorlatinib, лорлатиниб , لورلاتينيب , 洛拉替尼 , PF-6463922

It’s only fair to share… Lorlatinib, PF-6463922 For Cancer; Non-small-cell lung cancer Molecular Formula C21H19FN6O2 Average mass 406.413 Da Phase 2 WO 2013132376 Andrew James Jensen, Suman Luthra, Paul Francis RICHARDSON Applicant Pfizer Inc. (10R)-7-amino-12-fluoro-2,10,16-trimethyl-15-oxo-10,15,16,17-tetrahydro-2H-4,8- methenopyrazolo[4,3-h][2,5,11]benzoxadiazacyclotetradecine-3-carbonitrile (16R)-19-Amino-13-fluoro-4,8,16-trimethyl-9-oxo-17-oxa-4,5,8,20-tetraazatetracyclo[16.3.1.02,6.010,15]docosa-1(22),2,5,10,12,14,18,20-octaene-3-carbonitrile (10R)-7-Amino-12-fluoro-2,10,16-trimethyl-15-oxo-10,15,16,17-tetrahydro-2H-8,4-(metheno)pyrazolo[4,3-h][2,5,11]benzoxadiazacyclotetradecine-3-carbonitrile CAS 1454846-35-5 [RN] UNII:OSP71S83EU лорлатиниб [Russian] لورلاتينيب [Arabic] 洛拉替尼 [Chinese] Ros1 tyrosine kinase receptor inhibitor; Anaplastic lymphoma kinase receptor inhibitor useful …

Brigatinib, Бригатиниб, بريغاتينيب , 布格替尼 ,

It’s only fair to share… Brigatinib, AP26113 Molecular Formula: C29H39ClN7O2P Molecular Weight: 584.102 g/mol CAS 1197953-54-0 2,4-Pyrimidinediamine, 5-chloro-N4-[2-(dimethylphosphinyl)phenyl]-N2-[2-methoxy-4-[4-(4-methyl-1-piperazinyl)-1-piperidinyl]phenyl]- Бригатиниб[Russian][INN] بريغاتينيب[Arabic][INN] 布格替尼[Chinese][INN] 5-chloro-N4-[2-(dimethylphosphinyl)phenyl]-N2-[2-methoxy-4-[4-(4-methyl-1-piperazinyl)-1-piperidinyl]phenyl]-2,4-pyrimidinediamine AP-26113 MFCD29472221 UNII:HYW8DB273J In 2016, orphan drug designation was assigned to the compound in the U.S. for the treatment of ALK, ROS1 or EGFR-positive non-small cell lung cancer (NSCLC). WO 2009143389 PDT PAT Inventors Yihan …

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

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 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 …