Category «orphan drug»

Carglumic acid, карглумовая кислота , حمض كاروغلوميك , カルグルミ酸 ,

It’s only fair to share…   Carglumic acid N-Carbamyl-L-glutamate; Molecular FormulaC6H10N2O5 Average mass190.154 Da N-Carbamylglutamate карглумовая кислота [Russian] [INN] حمض كاروغلوميك [Arabic] [INN] カルグルミ酸; 1188-38-1 [RN] 5L0HB4V1EW 8008 L-Glutamic acid, N-(aminocarbonyl)- L-Glutamic acid, N-(hydroxyiminomethyl)- N-[Hydroxy(imino)methyl]-L-glutamic acid (S)-2-Ureidopentanedioic acid (S)-2-ureidopentanedioic acid; N-Carbamoyl-L-Glutamic Acid; N-Carbamyl-L-glutamate; N-Carbamylglutamate OE 312 / OE-312, UNII5L0HB4V1EW Prepn: H. McIlwain, Biochem. J. 33, 1942 (1939) Carglumic acid is a Carbamoyl Phosphate Synthetase 1 Activator. The mechanism …

Tecovirimat

It’s only fair to share… FDA approves the first drug with an indication for treatment of smallpox The U.S. Food and Drug Administration today approved TPOXX (tecovirimat), the first drug with an indication for treatment of smallpox. Though the World Health Organization declared smallpox, a contagious and sometimes fatal infectious disease, eradicated in 1980, there …

Isavuconazonium sulfate, Изавуконазониев сулфат

It’s only fair to share…   Isavuconazonium sulfate Изавуконазониев сулфат CAS 946075-13-4 MOLECULAR FORMULA: C35H36F2N8O9S2 MOLECULAR WEIGHT: 814.837 g/mol BAL-8557-002, BAL 8557 [2-[1-[1-[(2R,3R)-3-[4-(4-cyanophenyl)-1,3-thiazol-2-yl]-2-(2,5-difluorophenyl)-2-hydroxybutyl]-1,2,4-triazol-4-ium-4-yl]ethoxycarbonyl-methylamino]pyridin-3-yl]methyl 2-(methylamino)acetate;hydrogen sulfate UNII:31Q44514JV (2-{[(1-{1-[(2R,3R)-3-[4-(4-cyanophenyl)-1,3-thiazol-2-yl]-2-(2,5-difluorophenyl)-2-hydroxybutyl]-1H-1,2,4-triazol-4-ium-4-yl}ethoxy)carbonyl](methyl)amino}pyridin-3-yl)methyl N-methylglycinate hydrogen sulfate (2-{[(1-{1-[(2R,3R)-3-[4-(4-Cyanophenyl)-1,3-thiazol-2-yl]-2-(2,5-difluorophenyl)-2-hydroxybutyl]-1H-1,2,4-triazol-4-ium-4-yl}ethoxy)carbonyl](methyl)amino}-3-pyridinyl)methyl N-methylglycinate hydrog en sulfate FDA 2015, EU 2015, BAL8557-002, BCS CLASS I, RO-0098557 , AK-1820 fast track designation QIDP ORPHAN DRUG EU 1-{(2R,3R)-3-[4-(4-cyanophenyl)-1,3- thiazol-2-yl]-2-(2,5-difluoro-phenyl)-2-hydroxybutyl}-4-[(1RS)-1-({methyl[3-({[(methylamino)acetyl] oxy}methyl) pyridin-2-yl]carbamoyl}oxy)ethyl]-1H-1,2,4-triazol-4-ium monosulfate (IUPAC), …

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 …