Category «orphan drug»

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 …

ZY 19489, MMV 253

It’s only fair to share… ZY 19489, MMV 253 C24 H32 FN9, 465.5 CAS 1821293-40-6 MMV253, GTPL10024, MMV674253 N-(4-cyclopropyl-5-fluoro-6-methylpyridin-2-yl)-5-((3R)-2-((1,5-dimethyl-1H-pyrazol-3-yl)amino)-3,4-dimethylpiperazin-1-yl)pyrimidin-2-amine 2-N-(4-cyclopropyl-5-fluoro-6-methylpyridin-2-yl)-5-[(3R)-3,4-dimethylpiperazin-1-yl]-4-N-(1,5-dimethylpyrazol-3-yl)pyrimidine-2,4-diamine N2-(4-Cyclopropyl-5-fluoro-6-methyl-2-pyridinyl)-5-[(3R)-3,4-dimethyl-1-piperazinyl]-N4-(1,5-dimethyl-1H-pyrazol-3-yl)-2,4-pyrimidinediamine (R)-N2-(4-Cyclopropyl-5-fluoro-6-methylpyridin-2-yl)-N4-(1,5-dimethyl-1H-pyrazol-3-yl)-5-(3,4-dimethylpiperazin-1-yl)pyrimidine-2,4-diamine SYN IN 201721031453 The invention relates to triaminopyrimidine compd. of formula I, pharmaceutically acceptable salts thereof, hydrates, solvates, polymorphs, optically active forms thereof, in solid state forms useful for preventing or treating malaria.  The invention also relates …

Maribavir

It’s only fair to share…   Maribavir Molecular FormulaC15H19Cl2N3O4 Average mass376.235 Da FDA APROVED 11/23/2021, Livtencity 1263 W94, 1263W94 176161-24-3 [RN] 1H-Benzimidazol-2-amine, 5,6-dichloro-N-(1-methylethyl)-1-β-L-ribofuranosyl- UNII-PTB4X93HE1, марибавир , ماريبافير  ,马立巴韦 , BW-1263W94 Camvia, D04859, G1263, GW257406X 1263W94; BW-1263W94; GW-1263; GW-257406X; SHP-620; VP-41263 Company:GlaxoSmithKline (Originator) , Shire MOA:UL97 kinase inhibitor Indication:CMV prophylaxis To treat post-transplant cytomegalovirus (CMV) infection/disease that does not respond (with or without genetic mutations that cause resistance) to available antiviral …

Pafolacianine

It’s only fair to share…   Pafolacianine OTL-38 Molecular FormulaC61H67N9O17S4 Average mass1326.495 Da   2-{(E)-2-[(3E)-2-(4-{2-[(4-{[(2-Amino-4-oxo-3,4-dihydro-6-pteridinyl)methyl]amino}benzoyl)amino]-2-carboxyethyl}phenoxy)-3-{(2E)-2-[3,3-dimethyl-5-sulfo-1-(4-sulfobutyl)-1,3-dihydro-2H-indol-2-ylidene ]ethylidene}-1-cyclohexen-1-yl]vinyl}-3,3-dimethyl-1-(4-sulfobutyl)-3H-indolium-5-sulfonate OTL-38 Tyrosine, N-[4-[[(2-amino-3,4-dihydro-4-oxo-6-pteridinyl)methyl]amino]benzoyl]-O-[(6E)-6-[(2E)-2-[1,3-dihydro-3,3-dimethyl-5-sulfo-1-(4-sulfobutyl)-2H-indol-2-ylidene]ethylidene]-2-[(E)-2-[3,3-dimethy l-5-sulfo-1-(4-sulfobutyl)-3H-indolium-2-yl]ethenyl]-1-cyclohexen-1-yl]-, inner salt  2-(2-(2-(4-((2S)-2-(4-(((2-amino-4-oxo-3,4-dihydropteridin-6-yl)methyl)amino)benzamido)-2-carboxyethyl)phenoxy)-3-(2-(3,3-dimethyl-5-sulfo-1-(4-sulfobutyl)-1,3-dihydro-2H-indol-2-ylidene)ethylidene)cyclohex-1-en-1-yl)ethenyl)-3,3-dimethyl-5-sulfo-1-(4-sulfobutyl)-3H-indolium inner salt,sodium salt (1:4) 3H-Indolium, 2-(2-(2-(4-((2S)-2-((4-(((2-amino-3,4-dihydro-4-oxo-6-pteridinyl)methyl)amino)benzoyl)amino)-2-carboxyethyl)phenoxy)-3-(2-(1,3-dihydro-3,3-dimethyl-5-sulfo-1-(4-sulfobutyl)-2H-indol-2-ylidene)ethylidene)-1-cyclohexen-1-yl)ethenyl)-3,3-dimethyl-5-sulfo-1 (4-sulfobutyl)-, inner salt,sodium salt (1:4) 1628423-76-6 [RN] Pafolacianine sodium [USAN] RN: 1628858-03-6 UNII: 4HUF3V875C C61H68N9Na4O17S4+5 Intraoperative Imaging and Detection of Folate Receptor Positive Malignant Lesions Pafolacianine, sold under the …

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 …

Verdiperstat

It’s only fair to share… Verdiperstat AZD 3241; BHV-3241 CAS No. : 890655-80-8 1-(2-propan-2-yloxyethyl)-2-sulfanylidene-5H-pyrrolo[3,2-d]pyrimidin-4-one 4H-Pyrrolo[3,2-d]pyrimidin-4-one, 1,2,3,5-tetrahydro-1-[2-(1-methylethoxy)ethyl]-2-thioxo- 1-(2-isopropoxyethyl)-2-thioxo-1,2,3,5-tetrahydro-pyrrolo[3,2-d] pyrimidin-4-one l-(2-Isopropoxyethyl)-2-thioxo-l,2,3,5-tetrahydro-pyrrolo[3,2-d]pyrimidin-4-one Molecular FormulaC11H15N3O2S Average mass253.321 Da AZD-3241, BHV-3421, UNII-TT3345YXVR, TT3345YXVR, BHV-3241, WHO 10251 вердиперстат [Russian] [INN] فيرديبيرستات [Arabic] [INN] 维地泊司他 [Chinese] [INN] OriginatorAstraZeneca DeveloperAstraZeneca; Biohaven Pharmaceuticals ClassAntiparkinsonians; Ethers; Organic sulfur compounds; Pyrimidinones; Small molecules Mechanism of ActionPeroxidase inhibitors Orphan Drug StatusYes – Multiple system atrophy Phase IIIMultiple system …

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; …

Avalglucosidase alfa

It’s only fair to share… QQGASRPGPR DAQAHPGRPR AVPTQCDVPP NSRFDCAPDK AITQEQCEAR GCCYIPAKQG LQGAQMGQPW CFFPPSYPSY KLENLSSSEM GYTATLTRTT PTFFPKDILT LRLDVMMETE NRLHFTIKDP ANRRYEVPLE TPRVHSRAPS PLYSVEFSEE PFGVIVHRQL DGRVLLNTTV APLFFADQFL QLSTSLPSQY ITGLAEHLSP LMLSTSWTRI TLWNRDLAPT PGANLYGSHP FYLALEDGGS AHGVFLLNSN AMDVVLQPSP ALSWRSTGGI LDVYIFLGPE PKSVVQQYLD VVGYPFMPPY WGLGFHLCRW GYSSTAITRQ VVENMTRAHF PLDVQWNDLD YMDSRRDFTF NKDGFRDFPA MVQELHQGGR RYMMIVDPAI SSSGPAGSYR PYDEGLRRGV FITNETGQPL IGKVWPGSTA FPDFTNPTAL AWWEDMVAEF HDQVPFDGMW IDMNEPSNFI RGSEDGCPNN ELENPPYVPG VVGGTLQAAT …