Category «Orphan Drug Status»

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

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 https://www.fda.gov/downloads/BiologicsBloodVaccines/CellularGeneTherapyProducts/ApprovedProducts/UCM592766.pdf LUXTURNA STN: 125610 Proper Name: voretigene …

Elapegademase, エラペグアデマーゼ (遺伝子組換え)

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 Protein average weight 115000.0 Da …

Calaspargase pegol, カラスパルガーゼペゴル

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 APPROVED, Asparlas, FDA 2018/12/20 CAS 941577-06-6 UNII T9FVH03HMZ カラスパルガーゼペゴル; …

IMETELSTAT

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) 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), …

Tagraxofusp タグラクソフスプ

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 KLTFYLKTLE NAQAQQTTLS LAIF (disulfide bridge: …

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

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 the first therapeutic specifically indicated …

Icosapent ethyl, イコサペント酸エチル

Icosapent ethyl 330.5042 , C22H34O2 cas 86227-47-6 / 73310-10-8 ethyl (5Z,8Z,11Z,14Z,17Z)-icosa-5,8,11,14,17-pentaenoate Ethyl eicosapentaenoic acid イコサペント酸エチル (5Z,8Z,11Z,14Z,17Z)-Eicosapetaenoic acid ethyl ester (all-Z)-5,8,11,14,17-Eicosapentaenoic acid ethyl ester 5,8,11,14,17-Eicosapentaenoic acid, ethyl ester, (5Z,8Z,11Z,14Z,17Z)- [ACD/Index Name] 5,8,11,14,17-Eicosapentaenoic acid, ethyl ester, (all-Z)- 6GC8A4PAYH 86227-47-6 [RN] all-cis-5,8,11,14,17-Eicosapentaenoic Acid Ethyl Ester Timnodonic acid ethyl ester Vascepa 5,8,11,14,17-Eicosapentaenoic acid, ethyl ester, (all-Z)- (5Z,8Z,11Z,14Z,17Z)-5,8,11,14,17-Eicosapentaenoic acid ethyl ester (all-Z)-5,8,11,14,17-Eicosapentaenoic acid ethyl ester …

Tasimelteon, タシメルテオン

Tasimelteon N-([(1R,2R)-2-(2,3-Dihydro-1-benzofuran-4-yl)cyclopropyl]methyl)propanamide, 609799-22-6 [RN] 8985 Hetlioz [Trade name] N-{[(1R,2R)-2-(2,3-Dihydro-1-benzofuran-4-yl)cyclopropyl]methyl}propanamide [ACD/IUPAC Name] Propanamide, N-[[(1R,2R)-2-(2,3-dihydro-4-benzofuranyl)cyclopropyl]methyl]- [ACD/Index Name] SHS4PU80D9 609799-22-6 cas, BMS-214778; VEC-162, ATC:N05CH03 Use:Treatment of sleep disorder; Melatonin receptor agonist (1R,2R)-N-[2-(2,3-dihydrobenzofuran-4-yl)cyclopropylmethyl]propanamide Formula:C15H19NO2, MW:245.3 g/mol Hetlioz Vanda Pharmaceuticals, 2014 Approved fda 2014 EMA Tasimelteon is a white to off-white crystalline powder, it is non hygroscopic, soluble in water across relevant pH values and freely soluble …

FDA approves first drug Oxervate (cenegermin) for neurotrophic keratitis, a rare eye disease

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 the endoplasmic reticulum and cleaved …

Patisiran

Patisiran Sense strand: GUAACCAAGAGUAUUCCAUdTdT Anti-sense strand: AUGGAAUACUCUUGGUUACdTdT RNA, (A-U-G-G-A-A-Um-A-C-U-C-U-U-G-G-U-Um-A-C-dT-dT), complex with RNA (G-Um-A-A-Cm-Cm-A-A-G-A-G-Um-A-Um-Um-Cm-Cm-A-Um-dT-dT) (1:1), ALN-18328, 6024128 , ALN-TTR02 , GENZ-438027 , SAR-438037 , 50FKX8CB2Y (UNII code) for RNA, (A-U-G-G-A-A-Um-A-C-U-C-U-U-G-G-U-Um-A-C-dT-dT), complex with RNA(G-Um-A-A-Cm-Cm-A-A-G-A-G-Um-A-Um-Um-Cm-Cm-A-Um-dT-dT) (1:1) Nucleic Acid Sequence Sequence Length: 42, 21, 2112 a 7 c 7 g 4 t 12 umultistranded (2); modified CAS 1420706-45-1 Treatment of Amyloidosis, SEE…..https://endpts.com/gung-ho-alnylam-lands-historic-fda-ok-on-patisiran-revving-up-the-first-global-rollout-for-an-rnai-breakthrough/ Lipid-nanoparticle-encapsulated double-stranded siRNA targeting …