Category «Orphan Drug Status»

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), …

Macimorelin acetate

It’s only fair to share… Macimorelin Molecular FormulaC26H30N6O3 Average mass474.555 Da CAS  381231-18-1 Chemical Formula: C26H30N6O3 Exact Mass: 474.23794 Molecular Weight: 474.55480 Elemental Analysis: C, 65.80; H, 6.37; N, 17.71; O, 10.11 2-Methylalanyl-N-[(1R)-1-formamido-2-(1H-indol-3-yl)ethyl]-D-tryptophanamide 381231-18-1 [RN] 8680B21W73 9073 D-Tryptophanamide, 2-methylalanyl-N-[(1R)-1-(formylamino)-2-(1H-indol-3-yl)ethyl]- CAS 945212-59-9 (Macimorelin acetate) (2R)-2-(2-amino-2-methylpropanamido)-3-(1H-indol-3-yl)-N-[(1R)-2-(1H-indol-3-yl)-1-formamidoethyl]propanamide; acetic acid AEZS-130 ARD-07 D-87875 EP-01572 EP-1572 JMV-1843 USAN (ab-26) MACIMORELIN ACETATE AQZ1003RMG …

Caplacizumab-yhdp, カプラシズマブ

It’s only fair to share… FDA approves first therapy Cablivi (caplacizumab-yhdp) カプラシズマブ  , for the treatment of adult patients with a rare blood clotting disorder FDA February 6, 2019 Release https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm630851.htm?utm_campaign=020919_PR_FDA%20approves%20therapy%20for%20treatment%20of%20adult%20patients%20with%20rare%20blood%20clotting%20disorder&utm_medium=email&utm_source=Eloqua The U.S. Food and Drug Administration today approved Cablivi (caplacizumab-yhdp) injection, the first therapy specifically indicated, in combination with plasma exchange and immunosuppressive therapy, …

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) …

Tagraxofusp タグラクソフスプ

It’s only fair to share… MGADDVVDSS KSFVMENFSS YHGTKPGYVD SIQKGIQKPK SGTQGNYDDD WKGFYSTDNK YDAAGYSVDN ENPLSGKAGG VVKVTYPGLT KVLALKVDNA ETIKKELGLS LTEPLMEQVG TEEFIKRFGD GASRVVLSLP FAEGSSSVEY INNWEQAKAL SVELEINFET RGKRGQDAMY EYMAQACAGN RVRRSVGSSL SCINLDWDVI RDKTKTKIES LKEHGPIKNK MSESPNKTVS EEKAKQYLEE FHQTALEHPE LSELKTVTGT NPVFAGANYA AWAVNVAQVI DSETADNLEK TTAALSILPG IGSVMGIADG AVHHNTEEIV AQSIALSSLM VAQAIPLVGE LVDIGFAAYN FVESIINLFQ VVHNSYNRPA YSPGHKTRPH MAPMTQTTSL KTSWVNCSNM IDEIITHLKQ PPLPLLDFNN LNGEDQDILM ENNLRRPNLE AFNRAVKSLQ NASAIESILK NLLPCLPLAT AAPTRHPIHI KDGDWNEFRR …

Caplacizumab, カプラシズマブ Cablivi is the first therapeutic approved in Europe, for the treatment of a rare blood-clotting disorder

It’s only fair to share… Cablivi is the first therapeutic approved in Europe, for the treatment of a rare blood-clotting disorder On September 03, 2018, the European Commission has granted marketing authorization for Cablivi™ (caplacizumab) for the treatment of adults experiencing an episode of acquired thrombotic thrombocytopenic purpura (aTTP), a rare blood-clotting disorder. Cablivi is …