Tag «orphan drug»

Voretigene neparvovec , ボレチジーンネパルボベック;

It’s only fair to share… Voretigene neparvovec Voretigene neparvovec-rzyl; Luxturna (TN) ボレチジーンネパルボベック; DNA (synthetic adeno-associated virus 2 vector AAV2-hRPE65v2) CAS: 1646819-03-5 2017/12/19, FDA  Luxturna, SPARK THERAPEUTICS Vision loss treatment, Retinal dystrophy AAV2-hRPE65v2 AAV2.RPE65 LTW-888 SPK-RPE65 rAAV.hRPE65v2 rAAV2-CBSB-hRPE65 2SPI046IKD (UNII code) melting point (°C) 72-90ºC Rayaprolu V. et al. J. Virol. vol. 87. no. 24. (2013)   FDA …

Elapegademase, エラペグアデマーゼ (遺伝子組換え)

It’s only fair to share… AQTPAFNKPK VELHVHLDGA IKPETILYYG RKRGIALPAD TPEELQNIIG MDKPLSLPEF LAKFDYYMPA IAGSREAVKR IAYEFVEMKA KDGVVYVEVR YSPHLLANSK VEPIPWNQAE GDLTPDEVVS LVNQGLQEGE RDFGVKVRSI LCCMRHQPSW SSEVVELCKK YREQTVVAID LAGDETIEGS SLFPGHVKAY AEAVKSGVHR TVHAGEVGSA NVVKEAVDTL KTERLGHGYH TLEDTTLYNR LRQENMHFEV CPWSSYLTGA WKPDTEHPVV RFKNDQVNYS LNTDDPLIFK STLDTDYQMT KNEMGFTEEE FKRLNINAAK SSFLPEDEKK ELLDLLYKAY GMPSPA >>Elapegademase<<< AQTPAFNKPKVELHVHLDGAIKPETILYYGRKRGIALPADTPEELQNIIGMDKPLSLPEF LAKFDYYMPAIAGSREAVKRIAYEFVEMKAKDGVVYVEVRYSPHLLANSKVEPIPWNQAE GDLTPDEVVSLVNQGLQEGERDFGVKVRSILCCMRHQPSWSSEVVELCKKYREQTVVAID LAGDETIEGSSLFPGHVKAYAEAVKSGVHRTVHAGEVGSANVVKEAVDTLKTERLGHGYH TLEDTTLYNRLRQENMHFEVCPWSSYLTGAWKPDTEHPVVRFKNDQVNYSLNTDDPLIFK STLDTDYQMTKNEMGFTEEEFKRLNINAAKSSFLPEDEKKELLDLLYKAYGMPSPA Elapegademase, エラペグアデマーゼ (遺伝子組換え) EZN-2279 Protein chemical formula C1797H2795N477O544S12 …

Calaspargase pegol, カラスパルガーゼペゴル

It’s only fair to share… LPNITILATG GTIAGGGDSA TKSNYTAGKV GVENLVNAVP QLKDIANVKG EQVVNIGSQD MNDDVWLTLA KKINTDCDKT DGFVITHGTD TMEETAYFLD LTVKCDKPVV MVGAMRPSTS MSADGPFNLY NAVVTAADKA SANRGVLVVM NDTVLDGRDV TKTNTTDVAT FKSVNYGPLG YIHNGKIDYQ RTPARKHTSD TPFDVSKLNE LPKVGIVYNY ANASDLPAKA LVDAGYDGIV SAGVGNGNLY KTVFDTLATA AKNGTAVVRS SRVPTGATTQ DAEVDDAKYG FVASGTLNPQ KARVLLQLAL TQTKDPQQIQ QIFNQY (tetramer; disulfide bridge 77-105, 77′-105′, 77”-105”, 77”’-105”’)   Calaspargase pegol Molecular Formula, C1516-H2423-N415-O492-S8 (peptide monomer), Molecular Weight, 10261.2163 …

IMETELSTAT

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) …

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 …