It’s only fair to share… Danavorexton, TAK 925 2114324-48-8 Molecular FormulaC21H32N2O5S Average mass424.554 Da 1-Piperidinecarboxylic acid, 3-[(methylsulfonyl)amino]-2-[[(cis-4-phenylcyclohexyl)oxy]methyl]-, methyl ester, (2R,3S)- Methyl (2R,3S)-3-[(methylsulfonyl)amino]-2-[[(cis-4-phenylcyclohexyl)oxy]methyl]-1-piperidinecarboxylate OriginatorTakeda ClassCyclohexanes; Esters; Ethers; Piperidines; Sleep disorder therapies; Small molecules; Sulfonamides Mechanism of ActionOrexin receptor type 2 agonists Orphan Drug StatusYes – Narcolepsy Phase IHypersomnia; Narcolepsy; Respiration disorders; Sleep apnoea syndrome 01 …
Tag «orphan drug»
Darinaparsin
It’s only fair to share… SVG Image IUPAC Condensed H-gGlu-Cys(Unk)-Gly-OH Sequence XXG Darinaparsin ダリナパルシン , Darvias JAPAN 2022 APPROVED, PMDA 2022/6/20 (2S)-2-amino-5-[[(2R)-1-(carboxymethylamino)-3-dimethylarsanylsulfanyl-1-oxopropan-2-yl]amino]-5-oxopentanoic acid (S)-2-amino-5-(((R)-1-((carboxymethyl)amino)-3-((dimethylarsino)thio)-1-oxopropan-2-yl)amino)-5-oxopentanoic acid Glycine, L-gamma-glutaMyl-S-(diMethylarsino)-L-cysteinyl- Formula C12H22AsN3O6S CAS 69819-86-9 Mol weight 411.3062 Efficacy Antineoplastic Comment organic arsenical Zinapar, ZIO-101, DMAs(III)G, clarinaparsin, UNII-9XX54M675G, SP-02L OriginatorTexas A&M University; University of Texas M. D. Anderson Cancer Center DeveloperSolasia Pharma; ZIOPHARM Oncology ClassAmines; Antineoplastics; …
Olipudase alfa
It’s only fair to share… HPLSPQGHPA RLHRIVPRLR DVFGWGNLTC PICKGLFTAI NLGLKKEPNV ARVGSVAIKL CNLLKIAPPA VCQSIVHLFE DDMVEVWRRS VLSPSEACGL LLGSTCGHWD IFSSWNISLP TVPKPPPKPP SPPAPGAPVS RILFLTDLHW DHDYLEGTDP DCADPLCCRR GSGLPPASRP GAGYWGEYSK CDLPLRTLES LLSGLGPAGP FDMVYWTGDI PAHDVWHQTR QDQLRALTTV TALVRKFLGP VPVYPAVGNH ESTPVNSFPP PFIEGNHSSR WLYEAMAKAW EPWLPAEALR TLRIGGFYAL SPYPGLRLIS LNMNFCSREN FWLLINSTDP AGQLQWLVGE LQAAEDRGDK VHIIGHIPPG HCLKSWSWNY YRIVARYENT LAAQFFGHTH VDEFEVFYDE ETLSRPLAVA FLAPSATTYI GLNPGYRVYQ IDGNYSGSSH VVLDHETYIL NLTQANIPGA IPHWQLLYRA RETYGLPNTL …
PACRITINIB
It’s only fair to share… Pacritinib パクリチニブ; Formula C28H32N4O3 CAS 937272-79-2 Mol weight 472.5787 UPDATE FDA APPROVED 2/28/2022, Vonjo To treat intermediate or high-risk primary or secondary myelofibrosis in adults with low platelets A Jak2 inhibitor potentially for the treatment of acute myeloid Leukemia and myelofibrosis. UNII-G22N65IL3O пакритиниб باكريتينيب 帕瑞替尼 ONX-0803; SB-1518 CAS No. …
Efgartigimod alfa-fcab
It’s only fair to share… DKTHTCPPCP APELLGGPSV FLFPPKPKDT LYITREPEVT CVVVDVSHED PEVKFNWYVD GVEVHNAKTK PREEQYNSTY RVVSVLTVLH QDWLNGKEYK CKVSNKALPA PIEKTISKAK GQPREPQVYT LPPSRDELTK NQVSLTCLVK GFYPSDIAVE WESNGQPENN YKTTPPVLDS DGSFFLYSKL TVDKSRWQQG NVFSCSVMHE ALKFHYTQKS LSLSPGK (Disulfide bridge: 6-6′, 9-9′, 41-101, 147-205, 41′-101′, 147′-205′) Efgartigimod alfa-fcab Formula C2310H3554N602O692S14 CAS 1821402-21-4 Mol weight 51279.464 US FDA APPROVED 12/17/2021, To treat generalized myasthenia gravis Press Release, Vyvgart, BLA 761195 …
CILENGITIDE
It’s only fair to share… IUPAC Condensed cyclo[Arg-Gly-Asp-D-Phe-N(Me)Val] HELM PEPTIDE1{R.G.D.[dF].[meV]}$PEPTIDE1,PEPTIDE1,5:R2-1:R1$$$ IUPAC cyclo[L-arginyl-glycyl-L-alpha-aspartyl-D-phenylalanyl-N-methyl-L-valyl] CILENGITIDE Molecular FormulaC27H40N8O7 Average mass588.656 Da 2-[(2S,5R,8S,11S)-5-benzyl-11-[3-(diaminomethylideneamino)propyl]-7-methyl-3,6,9,12,15-pentaoxo-8-propan-2-yl-1,4,7,10,13-pentazacyclopentadec-2-yl]acetic acid 188968-51-6 [RN] 4EDF46E4GI 7823 циленгитид سيلانجيتيد 西仑吉肽 EMD 121974, EMD-121974, UNII-4EDF46E4GI 2-[(2S,5R,8S,11S)-5-benzyl-11-[3-(diaminomethylideneamino)propyl]-7-methyl-3,6,9,12,15-pentaoxo-8-propan-2-yl-1,4,7,10,13-pentazacyclopentadec-2-yl]acetic acid Cilengitide has been in phase III clinical trials by Merck Serono and NCI for the treatment of glioblastoma multiforme. However, this research has been …
Vosoritide
It’s only fair to share… PGQEHPNARK YKGANKKGLS KGCFGLKLDR IGSMSGLGC (Disulfide bridge: 23-39) H-Pro-Gly-Gln-Glu-His-Pro-Asn-Ala-Arg-Lys-Tyr-Lys-Gly-Ala-Asn-Lys-Lys-Gly-Leu-Ser-Lys-Gly-Cys(1)-Phe-Gly-Leu-Lys-Leu-Asp-Arg-Ile-Gly-Ser-Met-Ser-Gly-Leu-Gly-Cys(1)-OH PGQEHPNARKYKGANKKGLSKGCFGLKLDRIGSMSGLGC H-PGQEHPNARKYKGANKKGLSKGC(1)FGLKLDRIGSMSGLGC(1)-OH PEPTIDE1{P.G.Q.E.H.P.N.A.R.K.Y.K.G.A.N.K.K.G.L.S.K.G.C.F.G.L.K.L.D.R.I.G.S.M.S.G.L.G.C}$PEPTIDE1,PEPTIDE1,23:R3-39:R3$$$ L-prolyl-glycyl-L-glutaminyl-L-alpha-glutamyl-L-histidyl-L-prolyl-L-asparagyl-L-alanyl-L-arginyl-L-lysyl-L-tyrosyl-L-lysyl-glycyl-L-alanyl-L-asparagyl-L-lysyl-L-lysyl-glycyl-L-leucyl-L-seryl-L-lysyl-glycyl-L-cysteinyl-L-phenylalanyl-glycyl-L-leucyl-L-lysyl-L-leucyl-L-alpha-aspartyl-L-arginyl-L-isoleucyl-glycyl-L-seryl-L-methionyl-L-seryl-glycyl-L-leucyl-glycyl-L-cysteine (23->39)-disulfide (4R,10S,16S,19S,22S,28S,31S,34S,37S,40S,43S,49S,52R)-52-[[2-[[(2S)-6-amino-2-[[(2S)-2-[[(2S)-2-[[2-[[(2S)-6-amino-2-[[(2S)-6-amino-2-[[(2S)-4-amino-2-[[(2S)-2-[[2-[[(2S)-6-amino-2-[[(2S)-2-[[(2S)-6-amino-2-[[(2S)-2-[[(2S)-2-[[(2S)-4-amino-2-[[(2S)-1-[(2S)-2-[[(2S)-2-[[(2S)-5-amino-5-oxo-2-[[2-[[(2S)-pyrrolidine-2-carbonyl]amino]acetyl]amino]pentanoyl]amino]-4-carboxybutanoyl]amino]-3-(1H-imidazol-5-yl)propanoyl]pyrrolidine-2-carbonyl]amino]-4-oxobutanoyl]amino]propanoyl]amino]-5-carbamimidamidopentanoyl]amino]hexanoyl]amino]-3-(4-hydroxyphenyl)propanoyl]amino]hexanoyl]amino]acetyl]amino]propanoyl]amino]-4-oxobutanoyl]amino]hexanoyl]amino]hexanoyl]amino]acetyl]amino]-4-methylpentanoyl]amino]-3-hydroxypropanoyl]amino]hexanoyl]amino]acetyl]amino]-40-(4-aminobutyl)-49-benzyl-28-[(2S)-butan-2-yl]-31-(3-carbamimidamidopropyl)-34-(carboxymethyl)-16,22-bis(hydroxymethyl)-10,37,43-tris(2-methylpropyl)-19-(2-methylsulfanylethyl)-6,9,12,15,18,21,24,27,30,33,36,39,42,45,48,51-hexadecaoxo-1,2-dithia-5,8,11,14,17,20,23,26,29,32,35,38,41,44,47,50-hexadecazacyclotripentacontane-4-carboxylic acid Vosoritide Formula C176H290N56O51S3 CAS 1480724-61-5 Mol weight 4102.7254 1480724-61-5 [RN] BMN 111 L-Cysteine, L-prolylglycyl-L-glutaminyl-L-α-glutamyl-L-histidyl-L-prolyl-L-asparaginyl-L-alanyl-L-arginyl-L-lysyl-L-tyrosyl-L-lysylglycyl-L-alanyl-L-asparaginyl-L-lysyl-L-lysylglycyl-L-leucyl-L-seryl-L-lysylglycyl-L-cysteinyl-L-phenylalanylglycyl-L-leucyl-L-lysyl-L-leucyl-L-α-aspartyl-L-arginyl-L-isoleucylglycyl-L-seryl-L-methionyl-L-serylglycyl-L-leucylglycyl-, cyclic (23→39)-disulfide L-prolylglycyl-(human C-type natriuretic peptide-(17-53)-peptide (CNP-37)), cyclic-(23-39)-disulfide UNII:7SE5582Q2P восоритид [Russian] [INN] فوسوريتيد [Arabic] [INN] 伏索利肽 [Chinese] [INN] Voxzogo, 2021/8/26 EU APPROVED Product details Name Voxzogo Agency product number …
MAX 40279
It’s only fair to share… MAX 40279, EX-A4057 Max 4; MAX-40279; MAX-40279-001; MAX-40279-01 UNII-DL772G3NN7 2070931-57-4 C22H23FN6OS, 438.5 7-(4-fluoro-2-methoxyphenyl)-6-methyl-N-(1-piperidin-4-ylpyrazol-4-yl)thieno[3,2-d]pyrimidin-2-amine Thieno[3,2-d]pyrimidin-2-amine, 7-(4-fluoro-2-methoxyphenyl)-6-methyl-N-[1-(4-piperidinyl)-1H-pyrazol-4-yl]- 7-(4-FLUORO-2-METHOXYPHENYL)-6-METHYL-N-(1-(PIPERIDIN-4-YL)-1H-PYRAZOL-4-YL) THIENO (3,2-D)PYRIMIDIN-2-AMINE SEMI-FUMARATE CAS 2388506-43-0 7-(4-Fluoro-2-methoxyphenyl)-6-methyl-N-[1-(4-piperidinyl)-1H-pyrazol-4-yl]thieno[3,2-d]pyrimidin-2-amine Originator Maxinovel Pharmaceuticals ClassAntineoplastics Mechanism of ActionFibroblast growth factor receptor antagonists; Fms-like tyrosine kinase 3 inhibitors Orphan Drug StatusYes – Acute myeloid leukaemia Phase IAcute myeloid leukaemia; Solid tumours Most Recent …
Lonapegsomatropin
It’s only fair to share… FPTIPLSRLF DNAMLRAHRL HQLAFDTYQE FEEAYIPKEQ KYSFLQNPQT SLCFSESIPT PSNREETQQK SNLELLRISL LLIQSWLEPV QFLRSVFANS LVYGASDSNV YDLLKDLEEG IQTLMGRLED GSPRTGQIFK QTYSKFDTNS HNDDALLKNY GLLYCFRKDM DKVETFLRIV QCRSVEGSCG F (Disulfide bridge: 53-165, 182-189) Lonapegsomatropin, ロナペグソマトロピン FDA APPROVED, 25/8/21, Skytrofa, Treatment of growth hormone deficiency To treat short stature due to inadequate secretion of endogenous growth hormone 1934255-39-6 CAS, UNII: OP35X9610Y Molecular Formula, C1051-H1627-N269-O317-S9[-C2-H4-O]4n ACP 001; …
Nanatinostat
It’s only fair to share… Nanatinostat Tractinostat CHR-3996, CHR 3996, VRx 3996, C20H19FN6O2, 394.41 CAS 1256448-47-1 2-[(1α,5α,6α)-6-[[(6-Fluoro-2-q 2-[(1R,5S,6R)-6-{[(6-fluoroquinolin-2-yl)methyl]amino}-3-azabicyclo[3.1.0]hexan-3-yl]-N-hydroxypyrimidine-5-carboxamide 2-[(1R,5S,6s)-6-{[(6-Fluoro-2-quinolinyl)methyl]amino}-3-azabicyclo[3.1.0]hex-3-yl]-N-hydroxy-5-pyrimidinecarboxamide 5-Pyrimidinecarboxamide, 2-[(1R,5S)-6-[[(6-fluoro-2-quinolinyl)methyl]amino]-3-azabicyclo[3.1.0]hex-3-yl]-N-hydroxy- Chroma Therapeutics Ltd. (Originator) OriginatorChroma Therapeutics DeveloperChroma Therapeutics; Viracta Therapeutics ClassAmides; Antineoplastics; Pyrimidines; Quinolines; Small molecules Mechanism of ActionHistone deacetylase inhibitors Orphan Drug StatusYes – Post-transplant lymphoproliferative disorder; Plasmablastic lymphoma; T-cell lymphoma Phase IILymphoma Phase I/IIMultiple myeloma …