It’s only fair to share… IMETELSTAT CAS 868169-64-6, N163L Molecular Formula, C148-H211-N68-O53-P13-S13, Molecular Weight, 4610.2379, Nucleic Acid Sequence Sequence Length: 135 a 1 c 4 g 3 tmodified DNA d(3′-amino-3′-deoxy-P-thio)(T-A-G-G-G-T-T-A-G-A-C-A-A) 5′-[O-[2-hydroxy-3-[(1-oxohexadecyl)amino]propyl] hydrogen phosphorothioate] PHASE 3, GERON, Myelodysplasia IMETELSTAT SODIUM CAS 1007380-31-5, GRN163L, GRN 163L Sodium Salt Molecular Formula: C148H198N68Na13O53P13S13 Molecular Weight: 4895.941 g/mol 5′-(O-(2-hydroxy-3-((1-oxohexadecyl)amino)propyl)phosphorothioate)-d(3′-amino-3′-deoxy-p-thio)(t-a-g-g-g-t-t-a-g-a-c-a-a), sodium salt (13) …
Tag «orphan drug»
Selexipag, セレキシパグ ,селексипаг , سيليكسيباق ,
It’s only fair to share… Selexipag Molecular FormulaC26H32N4O4S Average mass496.622 Da Selexipag, Uptravi 475086-01-2 CAS (C26H32N4O4S, Mr = 496.6 g/mol) A prostacyclin receptor (PGI2) agonist used to treat pulmonary arterial hypertension (PAH). NIPPON SHINYAKU….INNOVATOR セレキシパグ UNII-5EXC0E384L селексипаг [Russian] [INN] سيليكسيباق [Amharic] [INN] 2-{4-[(5,6-diphenylpyrazin-2-yl)(propan-2-yl)amino]butoxy}-N-(methylsulfonyl)acetamide 475086-01-2 [RN] 5EXC0E384L 9231 Acetamide, 2-[4-[(5,6-diphenyl-2-pyrazinyl)(1-methylethyl)amino]butoxy]-N-(methylsulfonyl)- Selexipag (brand name Uptravi) is a drug developed by Actelion for the treatment of pulmonary arterial hypertension (PAH). …
USFDA approval to Lumoxiti (moxetumomab pasudotoxtdfk) a new treatment for hairy cell leukemia
It’s only fair to share… USFDA approval to Lumoxiti is a new treatment for hairy cell leukemia On September 13, 2018, the U.S. Food and Drug Administration approved Lumoxiti (moxetumomab pasudotoxtdfk) injection for intravenous use for the treatment of adult patients with relapsed or refractory Hairy Cell Leukemia (HCL) who have received at least two …
FDA approves first drug Oxervate (cenegermin) for neurotrophic keratitis, a rare eye disease
It’s only fair to share… http://www.who.int/medicines/publications/druginformation/issues/77_INN_Recommended_List.pdf Active Substance General information The active substance in Oxervate, cenegermin, is a recombinant human Nerve Growth factor (rhNGF) produced in E. coli strain HMS174. The molecule is identical to human Nerve Growth factor (NGF), a naturally occurring human protein. In humans, NGF is naturally produced as pre-pro-peptide, secreted into …
FDA approves first treatment Azedra (iobenguane I 131) for rare adrenal tumors
It’s only fair to share… Iobenguane I 131 FDA approves first treatment for rare adrenal tumors The U.S. Food and Drug Administration today approved Azedra (iobenguane I 131) injection for intravenous use for the treatment of adults and adolescents age 12 and older with rare tumors of the adrenal gland (pheochromocytoma or paraganglioma) …
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 NMR http://file.selleckchem.com/downloads/nmr/S854001-GBT440-CDCl3-hnmr-selleck.pdf 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 …
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 …