Tag «orphan drug»

FDA approves treatment Inrebic (fedratinib) for patients with rare bone marrow disorder

It’s only fair to share… FDA approves treatment Inrebic (fedratinib) for patients with rare bone marrow disorder Today, the U.S. Food and Drug Administration approved Inrebic (fedratinib) capsules to treat adult patients with certain types of myelofibrosis. “Prior to today, there was one FDA-approved drug to treat patients with myelofibrosis, a rare bone marrow disorder. …

Tanzisertib

It’s only fair to share… Tanzisertib CAS 899805-25-5 trans-4-((9-((3S)-Tetrahydrofuran-3-yl)-8-((2,4,6-trifluorophenyl)amino)-9H-purin-2-yl)amino)cyclohexanol 4-[[9-[(3S)-oxolan-3-yl]-8-(2,4,6-trifluoroanilino)purin-2-yl]amino]cyclohexan-1-ol C21-H23-F3-N6-O2, 448.4467 9557 Cyclohexanol, 4-[[9-[(3S)-tetrahydro-3-furanyl]-8-[(2,4,6-trifluorophenyl)amino]-9H-purin-2-yl]amino]-, trans- CC 930 CC-930 Tanzisertib UNII-M5O06306UO A c-Jun amino-terminal kinase inhibitor.UNII, M5O06306UO Treatment of Idiopathic Pulmonary Fibrosis (IPF) Originator Celgene Corporation Class Antifibrotics; Small molecules Mechanism of ActionJ NK mitogen-activated protein kinase inhibitors Orphan Drug Status Yes – Idiopathic pulmonary fibrosis Discontinued Discoid lupus erythematosus; …

Solriamfetol hydrochloride, ソルリアムフェトル塩酸塩 , солриамфетол , سولريامفيتول , 索安非托 ,

It’s only fair to share… Solriamfetol hydrochloride FDA APPROVED 2019/3/20, Sunosi ソルリアムフェトル塩酸塩; R228060, R 228060 Formula C10H14N2O2. HCl CAS 178429-65-7 HCL Mol weight 230.6913 (2R)-2-Amino-3-phenylpropyl carbamate (2R)-2-Amino-3-phenylpropylcarbamat 10117 178429-62-4 [RN] FREE FORM Benzenepropanol, β-amino-, carbamate (ester), (βR)- [ солриамфетол [Russian] [INN] سولريامفيتول [Arabic] [INN] 索安非托 [Chinese] [INN] JZP-110 Originator SK Holdings Developer Jazz Pharmaceuticals plc; SK biopharmaceuticals Class Carbamates; Sleep disorder therapies; Small molecules Mechanism of Action Adrenergic uptake inhibitors; Dopamine uptake …

RISDIPLAM , リスジプラム

It’s only fair to share… RISDIPLAM RG-7916, RO-7034067, リスジプラム Formula C22H23N7O Cas 1825352-65-5 Mol weight 401.4643 US9969754 7-(4,7-diazaspiro[2.5]octan-7-yl)-2-(2,8-dimethylimidazo[1,2-b]pyridazin-6-yl)pyrido[1,2-a]pyrimidin-4-one   WHO 10614 RG-7916 HY-109101 RO7034067 CS-0039501 EX-A2074 RG7916 The compound was originally claimed in WO2015173181 , for treating spinal muscular atrophy (SMA). Roche , under license from PTC Therapeutics , and Chugai , are developing risdiplam (RO-7034067; RG-7916), …

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 …