Tag «orphan drug»

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 …

VOXELOTOR

It’s only fair to share… VOXELOTOR GBT 440; GTx-011, Treatment of Sickle Cell Disease RN: 1446321-46-5 UNII: 3ZO554A4Q8 Molecular Formula, C19-H19-N3-O3, Molecular Weight, 337.3771 Benzaldehyde, 2-hydroxy-6-((2-(1-(1-methylethyl)-1H-pyrazol-5-yl)-3-pyridinyl)methoxy)- 2-hydroxy-6-((2-(1-(propan-2-yl)-1H-pyrazol-5-yl)pyridin-3-yl)methoxy)benzaldehyde Originator Global Blood Therapeutics Class Antianaemics; Small molecules Mechanism of Action Abnormal haemoglobin modulators; Sickle haemoglobin modulators Orphan Drug Status Yes – Sickle cell anaemia New Molecular Entity Yes Highest Development Phases Phase III Sickle cell anaemia Phase …

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 …