Category «Orphan Drug Status»

Chenodeoxycholic acid, ケノデオキシコール酸

It’s only fair to share… Chenodeoxycholic acid Chenodiol Molecular FormulaC24H40O4 Average mass392.572 UNII-0GEI24LG0J ケノデオキシコール酸 474-25-9 [RN] chenodeoxycholic acid [JP15] [Wiki] (+)-chenodeoxycholic acid (3a,5b,7a)-3,7-dihydroxy-cholan-24-oic acid (3α,5β,7α,8ξ,20R)-3,7-Dihydroxycholan-24-säure[German] [ACD/IUPAC Name] (4R)-4-[(3R,5S,7R,8R,9S,10S,13R,14S,17R)-3,7-Dihydroxy-10,13-dimethylhexadecahydro-1H-cyclopenta[a]phenanthren-17-yl]pentanoic acid 0GEI24LG0J 17b-(1-Methyl-3-carboxypropyl)etiocholane-3a,7a-diol 207-481-8[EINECS] 3a,7a-dihydroxy-5b-cholan-24-oic acid 3a,7a-dihydroxy-5b-cholanic acid; anthropodesoxycholic acid; gallodesoxycholic acid; 17b-(1-methyl-3-carboxypropyl)etiocholane-3a,7a-diol; chenic acid; chenodeoxycholic acid; CDC Chenodeoxycholate; Chenodeoxycholic acid; 3alpha,7alpha-Dihydroxy-5beta-cholanic acid; Chenodiol Synthesis ReferenceHenry Francis Frost, Fritz Fabian, Christopher James …

RG7440, Ipatasertib, アイパタセルチブ;

It’s only fair to share…   Ipatasertib GDC-0068 , RG7440 CAS 1001264-89-6, C24H32ClN5O2, 457.9962 アイパタセルチブ; イパタセルチブ; Antineoplastic, AKT serine/threonine kinase inhibitor 2(S)-(4-Chlorophenyl)-1-[4-[7(R)-hydroxy-5(R)-methyl-6,7-dihydro-5H-cyclopenta[d]pyrimidin-4-yl]piperazin-1-yl]-3-(isopropylamino)propan-1-one (2S)-2-(4-Chlorophenyl)-1-(4-((5R,7R)-7-hydroxy-5-methyl-6,7-dihydro-5H-cyclopenta(d)pyrimidin-4-yl)piperazin-1-yl(-3-((propan-2-yl)amino)propan-1-one 1-Propanone, 2-(4-chlorophenyl)-1-(4-((5R,7R)-6,7-dihydro-7-hydroxy-5-methyl-5H-cyclopentapyrimidin-4-yl)-1-piperazinyl)-3-((1-methylethyl)amino)-,  (2S)- Ipatasertib dihydrochloride 1396257-94-5 Ipatasertib (RG7440) is an experimental cancer drug in development by Roche. It is a small molecule inhibitor of Akt. It was discovered by Array Biopharma and is currently in phase II trials for treatment of breast …

Glasdegib, PF-04449913

It’s only fair to share…   Glasdegib (PF-04449913) 1-[(2R,4R)-2-(1H-Benzimidazol-2-yl)-1-methyl-4-piperidinyl]-3-(4-cyanophenyl)urea [ACD/IUPAC Name] 1-[(2R,4R)-2-(1H-benzimidazol-2-yl)-1-methylpiperidin-4-yl]-3-(4-cyanophenyl)urea CAS 1095173-27-5 [RN], Orphan Drug Status Glasdegib Molecular FormulaC21H22N6O Average mass374.439 Da Urea, N-[(2R,4R)-2-(1H-benzimidazol-2-yl)-1-methyl-4-piperidinyl]-N’-(4-cyanophenyl)- [ACD/Index Name] гласдегиб [Russian] [INN] غلاسديغيب [Arabic] [INN] 格拉德吉 [Chinese] [INN] FACT SHEET   https://www.pfizer.com/files/news/asco/Glasdegib-Fact-Sheet-6JUNE2018.pdf Glasdegib (PF-04449913) is an experimental cancer drug developed by Pfizer. It is a small molecule inhibitor of the Sonic hedgehog pathway, which is overexpressed in many types of …

TIPIFARNIB, типифарниб , تيبيفارنيب , 替匹法尼 ,

It’s only fair to share… TIPIFARNIB R-115777, NSC-702818 Categories Antineoplastic Agents P-glycoprotein/ABCB1 Inhibitors Quinolines UNIIMAT637500A CAS number 192185-72-1 +form 192185-68-5 (racemate) 192185-69-6 (racemic; fumarate) 192185-70-9 (racemic; diHCl) (+)-(R)-6-[1-Amino-1-(4-chlorophenyl)-1-(1-methylimidazol-5-yl)methyl]-4-(3-chlorophenyl)-1-methylquinolin-2(1H)-one 2(1H)-Quinolinone, 6-[(R)-amino(4-chlorophenyl)(1-methyl-1H-imidazol-5-yl)methyl]-4-(3-chlorophenyl)-1-methyl- Weight Average: 489.396 Chemical Formula C27H22Cl2N4O типифарниб [Russian] [INN] تيبيفارنيب [Arabic] [INN] 替匹法尼 [Chinese] [INN] (R)-(+)-R115777 (R)-6-(Amino(4-chlorophenyl)(1-methyl-1H-imidazol-5-yl)methyl)-4-(3-chlorophenyl)-1-methyl-2(1H)-quinolinone (R)-6-(amino(4-chlorophenyl)(1-methyl-1H-imidazol-5-yl)methyl)-4-(3-chlorophenyl)-1-methylquinolin-2(1H)-one 2 (1H))-Quinolinone,6-(amino(4-chlorophenyl)(1-methyl-1H-imidazol-5-yl)methyl)-4-(3-chlorophenyl)-1-methyl-, 2(1H )-quinolinone Title: Tipifarnib CAS Registry Number: 192185-72-1; 192185-68-5 (unspecified stereo) CAS Name: 6-[(R)-Amino(4-chlorophenyl)(1-methyl-1H-imidazol-5-yl)methyl]-4-(3-chlorophenyl)-1-methyl-2(1H)-quinolinone Manufacturers’ Codes: R-115777 Trademarks: Zarnestra …

Fedratinib

It’s only fair to share… FEDRATINIB SAR-302503; TG-101348, 6L1XP550I6, 936091-26-8 [RN], WHO 9707 Molecular Formula: C27H36N6O3S Molecular Weight: 524.684 g/mol FLT3, JAK2 http://www.ama-assn.org//resources/doc/usan/fedratinib.pdf Fedratinib had been in phase III clincial trials by Sanofi for the treatment of myelofibrosis. However, Sanofi had discontinued this research because of the safety issues. Orphan drug designation was assigned in the U.S. and in …

Larotrectinib, ларотректиниб , 拉罗替尼 ,

It’s only fair to share…   Larotrectinib ARRY-470, LOXO-101, PF9462I9HX Molecular Formula: C21H22F2N6O2 Molecular Weight: 428.444 g/mol (3S)-N-{5-[(2R)-2-(2,5-Difluorphenyl)-1-pyrrolidinyl]pyrazolo[1,5-a]pyrimidin-3-yl}-3-hydroxy-1-pyrrolidincarboxamid (S)-N-{5-[(R)-2-(2,5-Difluorophenyl)pyrrolidin-1-yl]pyrazolo[1,5-a]pyrimidin-3-yl}-3-hydroxypyrrolidine-1-carboxamide 10360 1223403-58-4 [RN] UNII:PF9462I9HX ларотректиниб [Russian] [INN] 拉罗替尼 [Chinese] [INN] (3S)-N-[5-[(2R)-2-(2,5-difluorophenyl)pyrrolidin-1-yl]pyrazolo[1,5-a]pyrimidin-3-yl]-3-hydroxypyrrolidine-1-carboxamide NTRK-fusion solid tumours TRK inhibitor orphan drug designation in the U.S In 2013, Array Biopharma licensed the product to Loxo Oncology for development and commercialization in the U.S. In 2016, breakthrough therapy …

ELECLAZINE, элеклазин , إيليكلازين , 依来克秦 , REVISITED

It’s only fair to share… ELECLAZINE GS-6615 Molecular Formula: C21H16F3N3O3 Molecular Weight: 415.372 g/mol 1443211-72-0 4-(pyrimidin-2-ylmethyl)-7-[4-(trifluoromethoxy)phenyl]-2,3-dihydro-1,4-benzoxazepin-5-one 4-(pyrimidin-2-ylmethyl)-7-[4-(trifluoromethoxy)phenyl]-2,3,4,5- tetrahydro-1,4- benzoxazepin-5-one 7-(4-(Trifluoromethoxy)phenyl)-3,4-dihydrobenzo[f][1,4]oxazepin-5(2H)-one 1,4-Benzoxazepin-5(2H)-one, 3,4-dihydro-4-(2-pyrimidinylmethyl)-7-[4-(trifluoromethoxy)phenyl]- Eleclazine; UNII-PUY08529FK; 1443211-72-0; GS-6615; PUY08529FK; 4-(pyrimidin-2-ylmethyl)-7-(4-(trifluoromethoxy)phenyl)-3,4-dihydrobenzo[f][1,4]oxazepin-5(2H)-on элеклазин [Russian] [INN] إيليكلازين [Arabic] [INN] 依来克秦 [Chinese] [INN] Phase III Long QT syndrome INGREDIENT UNII CAS Eleclazine Hydrochloride 4R1JP3Q4HI 1448754-43-5 Eleclazine has been used in trials studying the treatment of LQT2 Syndrome, Long …

Acalabrutinib, ACP-196, Акалабрутиниб , أكالابروتينيب , 阿可替尼 ,

It’s only fair to share… Acalabrutinib Molecular FormulaC26H23N7O2 Average mass465.507 Da Acalabrutinib, rINN, ACP-196, FDA 2017 APPROVED, Lymphoma, mantle cell, ACERTA PHARMA Orphan Drug, breakthrough therapy designation, CAS 1420477-60-6 [RN] (S)-4-[8-Amino-3-[1-(but-2-ynoyl)pyrrolidin-2-yl]imidazo[1,5-a]pyrazin-1-yl]-N-(pyridin-2-yl)benzamide (S)-4-(8-amino-3-n-but-2-vnoylpyrrolidin-2-vnimidazo[1 ,5-alpyrazin-1-yl)-N-(pyridin-2-yl)benzamide 4-{8-Amino-3-[(2S)-1-(2-butynoyl)-2-pyrrolidinyl]imidazo[1,5-a]pyrazin-1-yl}-N-(2-pyridinyl)benzamide Benzamide, 4-[8-amino-3-[(2S)-1-(1-oxo-2-butyn-1-yl)-2-pyrrolidinyl]imidazo[1,5-a]pyrazin-1-yl]-N-2-pyridinyl- Calquence [Trade name] UNII:I42748ELQW Акалабрутиниб [Russian] [INN] أكالابروتينيب [Arabic] [INN] 阿可替尼 [Chinese] [INN] 4-[8-amino-3-[(2S)-1-(1-oxo-2-butyn-1-yl)-2-pyrrolidinyl]imidazo[1,5-a]pyrazin-1-yl]-N-2-pyridinyl-benzamide 4-[8-amino-3-[(2S)-1-but-2-ynoylpyrrolidin-2-yl]imidazo[1,5-a]pyrazin-1-yl]-N-pyridin-2-ylbenzamide I42748ELQW  Acalabrutinib, also known as ACP-196, is an orally available inhibitor of Bruton’s tyrosine kinase (BTK) …

FDA approves first drug for Eosinophilic Granulomatosis with Polyangiitis, a rare disease formerly known as the Churg-Strauss Syndrome

It’s only fair to share… FDA approves first drug for Eosinophilic Granulomatosis with Polyangiitis, a rare disease formerly known as the Churg-Strauss Syndrome The U.S. Food and Drug Administration today expanded the approved use of Nucala (mepolizumab) to treat adult patients with eosinophilic granulomatosis with polyangiitis (EGPA), a rare autoimmune disease that causes vasculitis, an …

Upadacitinib, ABT-494, упадацитиниб , أوباداسيتينيب , 乌帕替尼 ,

It’s only fair to share… ABT 494 (-)-(3S,4R)  cis form CAS 1310726-60-3 FREE FORM    MF C17H19F3N6O MW 380.36757 g/mol Tartrate form  C17 H19 F3 N6 O . C4 H6 O6 . 4 H2 O ………….CAS 1607431-21-9 1-Pyrrolidinecarboxamide, 3-ethyl-4-(3H-imidazo[1,2-a]pyrrolo[2,3-e]pyrazin-8-yl)-N-(2,2,2-trifluoroethyl)-, (3S,4R)-, (2R,3R)-2,3-dihydroxybutanedioate, hydrate (1:1:4) FREE FORM (3s,,4R)-3-ethyl-4-(3H-imidazo[l,2-fl]pyrrolo[2,3-e]pyrazin-8-yl)-N-(2,2,2-trifluoroethyl)pyrrolidine- l-carboxamide. (35,,4R)-3-ethyl-4-(3H- imidazo[l,2-fl]pyrrolo[2,3-e]pyrazin-8-yl)-N-(2,2,2-trifluoroethyl)pyrrolidine- l-carboxamide, (cis,)-3-ethyl-4-(3H-imidazo[l,2-fl]pyrrolo[2,3-e]pyrazin-8-yl)-N-(2,2,2-trifluoroethyl)pyrrolidine-l-carboxamide 1-Pyrrolidinecarboxamide, 3-ethyl-4-(3H-imidazo[1,2-a]pyrrolo[2,3-e]pyrazin-8-yl)-N-(2,2,2-trifluoroethyl)-, (3S,4R)- rel-(-)-(3S,4R)-3-Ethyl-4-(3H-imidazo[1,2-a]pyrrolo[2,3-e]pyrazin-8-yl)-N-(2,2,2-trifluoroethyl)pyrrolidine-1-carboxamide A Jak1 …