Category «Orphan Drug Status»

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 …

Pracinostat

It’s only fair to share… Pracinostat Molecular Formula C20H30N4O2 Average mass 358.478 Da 2-Propenamide, 3-[2-butyl-1-[2-(diethylamino)ethyl]-1H-benzimidazol-5-yl]-N-hydroxy-, (2E)- 929016-96-6 [RN] SB939 (2E)-3-{2-butyl-1-[2-(diethylamino)ethyl]-1,3-benzodiazol-5-yl}-N-hydroxyprop-2-enamide N-hydroxy-1-[(4-methoxyphenyl)methyl]-1H-indole-6-carboxamide PCI 34051,  UNII: GPO2JN4UON 929016-98-8 DI HCl salt, C20 H30 N4 O2 . 2 Cl H, 431.4 929016-96-6 (free base) 929016-97-7 (trifluoroacetate) S*BIO (Originator) Leukemia, acute myeloid, phase 3, helsinn CAS 929016-98-8 DI HCl salt, C20 H30 N4 O2 . 2 Cl …

Funapide, TV 45070, XEN-402, фунапид فونابيد 呋纳匹特

It’s only fair to share… Funapide TV 45070,  XEN-402,  Funapide, (+)- фунапид فونابيد 呋纳匹特 Molecular FormulaC22H14F3NO5 Average mass429.345 Da (S)-1′-[(5-Methyl-2-furyl)methyl]spiro[6H-furo[3,2-f][1,3]benzodioxole-7,3′-indoline]-2′-one Spiro(furo(2,3-F)-1,3-benzodioxole-7(6H),3′-(3H)indol)-2′(1’H)-one, 1′-((5-(trifluoromethyl)-2-furanyl)methyl)-, (3’S)- (3’S)-1′-((5-(Trifluoromethyl)furan-2-yl)methyl)-2H,6H-spiro(furo(2,3-F)(1,3)benzodioxole-7,3′-indol)-2′(1’H)-one Spiro[furo[2,3-f]-1,3-benzodioxole-7(6H),3′-[3H]indol]-2′(1’H)-one, 1′-[[5-(trifluoromethyl)-2-furanyl]methyl]-, (7S)- TV-45070 UNII-A5595LHJ2L XEN-401-S XEN402 (3’S)-1′-{[5-(trifluoromethyl)furan-2-yl]methyl}-2H-6H-spiro[furo[2,3-f]-1,3-benzodioxole-7,3′-indol]-2′(1’H)-one (7S)-1′-{[5-(Trifluoromethyl)-2-furyl]methyl}spiro[furo[2,3-f][1,3]benzodioxole-7,3′-indol]-2′(1’H)-one 1259933-16-8 CAS UNII-A5595LHJ2L Phase II clinical trials for Postherpetic neuralgia (PHN) Treatment of Neuropathic Pain Originator Xenon Pharmaceuticals Developer Teva Pharmaceutical Industries; Xenon Pharmaceuticals Class Benzodioxoles; Fluorobenzenes; …

Enasidenib, Энасидениб , إيناسيدينيب ,伊那尼布 ,

It’s only fair to share…   Enasidenib Molecular Formula C19H17F6N7O Average mass 473.375 2-Propanol, 2-methyl-1-[[4-[6-(trifluoromethyl)-2-pyridinyl]-6-[[2-(trifluoromethyl)-4-pyridinyl]amino]-1,3,5-triazin-2-yl]amino]- 2-Methyl-1-[[4-[6-(trifluoromethyl)-2-pyridinyl]-6-[[2-(trifluoromethyl)-4-pyridinyl]amino]-1,3,5-triazin-2-yl]amino]-2-propanol 2-Methyl-1-(4-(6-(trifluoromethyl)pyridin-2-yl)-6-(2-(trifluoromethyl)pyridin-4-ylamino)-1,3,5-triazin-2-ylamino)propan-2-ol   AG-221 CC-90007 1446502-11-9[RN] enasidenib Enasidenib énasidénib enasidenibum UNII:3T1SS4E7AG Энасидениб[Russian] إيناسيدينيب[Arabic] 伊那尼布[Chinese] 2-methyl-1-[(4-[6-(trifluoromethyl)pyridin-2-yl]-6-{[2-(trifluoromethyl)pyridin-4-yl]amino}-1,3,5-triazin-2-yl)amino]propan-2-ol 2-methyl-1-[[4-[6-(trifluoromethyl)pyridin-2-yl]-6-[[2-(trifluoromethyl)pyridin-4-yl]amino]-1,3,5-triazin-2-yl]amino]propan-2-ol 2-methyl-1-(4-(6-(trifluoromethyl)pyridin-2-yl)-6-(2-(trifluoromethyl)pyridin-4-ylamino)-1,3,5-triazin-2-ylamino)propan-2-ol Originator Agios Pharmaceuticals Developer Celgene Corporation Mechanism Of Action Isocitrate dehydrogenase 2 inhibitor Who Atc Codes L01 (Antineoplastic Agents) Ephmra Codes L1 (Antineoplastics) Indication Cancer …

FDA approves drug to treat ALS, Radicava (Edaravone) , эдаравон, إيدارافون , 依达拉奉 ,ラジカット,

It’s only fair to share… FDA approves drug to treat ALS 05/05/2017 The U.S. Food and Drug Administration today approved Radicava (edaravone) to treat patients with amyotrophic lateral sclerosis (ALS), commonly referred to as Lou Gehrig’s disease. May 5, 2017 Release The U.S. Food and Drug Administration today approved Radicava (edaravone) to treat patients with …

FDA approves new combination treatment for acute myeloid leukemia, Rydapt (midostaurin)

It’s only fair to share… MIDOSTAURIN FDA approves new combination treatment for acute myeloid leukemia 04/28/2017 The U.S. Food and Drug Administration today approved Rydapt (midostaurin) for the treatment of adult patients with newly diagnosed acute myeloid leukemia (AML) who have a specific genetic mutation called FLT3, in combination with chemotherapy. The drug is approved …

FDA approves first treatment for a form of Batten disease, Brineura (cerliponase alfa)

It’s only fair to share… FDA approves first treatment for a form of Batten disease 04/27/2017 The U.S. Food and Drug Administration today approved Brineura (cerliponase alfa) as a treatment for a specific form of Batten disease. Brineura is the first FDA-approved treatment to slow loss of walking ability (ambulation) in symptomatic pediatric patients 3 …

BLU 285

It’s only fair to share… BLU-285 CAS 1703793-34-3 Molecular FormulaC26H27FN10 Average mass498.558 Da (1S)-1-(4-Fluorophenyl)-1-(2-{4-[6-(1-methyl-1H-pyrazol-4-yl)pyrrolo[2,1-f][1,2,4]triazin-4-yl]-1-piperazinyl}-5-pyrimidinyl)ethanamine 5-Pyrimidinemethanamine, α-(4-fluorophenyl)-α-methyl-2-[4-[6-(1-methyl-1H-pyrazol-4-yl)pyrrolo[2,1-f][1,2,4]triazin-4-yl]-1-piperazinyl]-, (αS)- 5-Pyrimidinemethanamine, α-(4-fluorophenyl)-α-methyl-2-[4-[6-(1-methyl-1H-pyrazol-4-yl)pyrrolo[2,1-f][1,2,4]triazin-4-yl]-1-piperazinyl]-, (αS)- Originator Blueprint Medicines Class Antineoplastics; Skin disorder therapies; Small molecules Mechanism of Action Platelet-derived growth factor alpha receptor modulators; Proto oncogene protein c-kit inhibitors Orphan Drug Status Yes – Systemic mastocytosis; Gastrointestinal stromal tumours Phase I Gastrointestinal stromal tumours; Solid tumours; …

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, …

FDA approves Xermelo (telotristat ethyl) for carcinoid syndrome diarrhea

It’s only fair to share…   Telotristat ethyl Molecular Formula, C27-H26-Cl-F3-N6-O3, Molecular Weight, 574.9884, RN: 1033805-22-9 UNII: 8G388563M LX 1032 (2S)-2-Amino-3-[4-[2-amino-6-[[(1R)-1-[4-chloro-2-(3-methylpyrazol-1-yl)phenyl]-2,2,2-trifluoroethyl]oxy]pyrimidin-4-yl]phenyl]propionic acid ethyl ester Ethyl-4-(2-amino-6-{(1R)-1-[4-chlor-2-(3-methyl-1H-pyrazol-1-yl)phenyl]-2,2,2-trifluorethoxy}-4-pyrimidinyl)-L-phenylalaninat L-Phenylalanine, 4-[2-amino-6-[(1R)-1-[4-chloro-2-(3-methyl-1H-pyrazol-1-yl)phenyl]-2,2,2-trifluoroethoxy]-4-pyrimidinyl]-, ethyl ester SEE…………… Telotristat etiprate, (S)-ethyl 2-amino-3-(4-(2-amino-6-((R)-1-(4-chloro-2-(3-methyl-1H-pyrazol-1-yl)phenyl)-2,2,2-trifluoroethoxy)pyrimidin-4-yl)phenyl)propanoate 2-benzamidoacetate . CAS: 1137608-69-5 (etiprate), LX 1606 Chemical Formula: C36H35ClF3N7O6 Molecular Weight: 754.16 L-Phenylalanine, 4-[2-amino-6-[(1R)-1-[4-chloro-2-(3-methyl-1H-pyrazol-1-yl)phenyl]-2,2,2-trifluoroethoxy]-4-pyrimidinyl]-, ethyl ester, compd. with N-benzoylglycine (1:1) LX 1032 …