orphan drug – Page 2 – Drug Approvals International

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 …

Piflufolastat F 18 injection, Dcfpyl F-18

It’s only fair to share… Piflufolastat F 18 injection Dcfpyl F-18 CAS 207181-29-0 PLAIN F 1423758-00-2 WITHOUT RADIO LABEL C18 H23 F N4 O8, 441.4 L-Glutamic acid, N-[[[(1S)-1-carboxy-5-[[[6-(fluoro-18F)-3-pyridinyl]carbonyl]amino]pentyl]amino]carbonyl]- 2-(3-{1-carboxy-5-[(6-[18F]fluoro-pyridine-3-carbonyl) amino]-pentyl}ureido)-pentanedioic acid Other Names N-[[[(1S)-1-Carboxy-5-[[[6-(fluoro-18F)-3-pyridinyl]carbonyl]amino]pentyl]amino]carbonyl]-L-glutamic acid [18F]DCFPyl Dcfpyl F-18 (18F)Dcfpyl UNII-3934EF02T7 18F-DCFPyL 3934EF02T7 Progenics Pharmaceuticals, Inc. APPROVED 5/26/2021 fda, Pylarify For positron emission tomography imaging of prostate-specific membrane antigen-positive lesions in men with prostate …

Sotorasib

It’s only fair to share… Sotorasib 6-fluoro-7-(2-fluoro-6-hydroxyphenyl)-1-(4-methyl-2-propan-2-ylpyridin-3-yl)-4-[(2S)-2-methyl-4-prop-2-enoylpiperazin-1-yl]pyrido[2,3-d]pyrimidin-2-one AMG 510 AMG-510 AMG510 Formula C30H30F2N6O3 CAS 2296729-00-3 Mol weight 560.5944 FDA APPROVED, 2021/5/28 Lumakras Antineoplastic, Non-small cell lung cancer (KRAS G12C-mutated) ソトラシブ (JAN); 2296729-00-3 (racemate) 4-((S)-4-Acryloyl-2-methylpiperazin-1-yl)-6-fluoro-7-(2-fluoro-6-hydroxyphenyl)-1-(2-isopropyl-4-methylpyridin-3-yl)pyrido[2,3-d]pyrimidin-2(1H)-one 6-fluoro-7-(2-fluoro-6-hydroxyphenyl)-1-(4-methyl-2-propan-2-ylpyridin-3-yl)-4-[(2S)-2-methyl-4-prop-2-enoylpiperazin-1-yl]pyrido[2,3-d]pyrimidin-2-one Sotorasib [INN] 6-Fluoro-7-(2-fluoro-6-hydroxyphenyl)-1-(4-methyl-2-propan-2-ylpyridin-3-yl)-4-((2S)-2-methyl-4-prop-2-enoylpiperazin-1-yl)pyrido(2,3-d)pyrimidin-2-one Sotorasib (1M)-6-Fluoro-7-(2-fluoro-6-hydroxyphenyl)-1-[4-methyl-2-(propan-2-yl)pyridin-3-yl]-4-[(2S)-2-methyl-4-(prop-2-enoyl)piperazin-1-yl]pyrido[2,3-d]pyrimidin-2(1H)-one C30H30F2N6O3 : 560.59 [2296729-00-3] Sotorasib is an inhibitor of the RAS GTPase family. The molecular formula is C30H30F2N6O3, and the …

Infigratinib phosphate

It’s only fair to share… Infigratinib phosphate FDA APPR Truseltiq 2021/5/28 インフィグラチニブリン酸塩; 3-(2,6-dichloro-3,5-dimethoxyphenyl)-1-[6-[4-(4-ethylpiperazin-1-yl)anilino]pyrimidin-4-yl]-1-methylurea;phosphoric acid BGJ 398 BGJ-398 BGJ398 NVP-BGJ398 WHO 10032 Product Ingredients INGREDIENT UNII CAS INCHI KEY Infigratinib acetate 03D0789NYP 1310746-17-8 XHCQHOGMMJKLRU-UHFFFAOYSA-N Infigratinib hydrochloride WY8VD4RV77 1310746-15-6 VBAIJSJSFCXDJB-UHFFFAOYSA-N Infigratinib mesylate E223Z0KWCC 1310746-12-3 BXJJFNXYWJLBOS-UHFFFAOYSA-N Infigratinib phosphate 58BH47BV6S 1310746-10-1 GUQNHCGYHLSITB-UHFFFAOYSA-N International/Other BrandsTruseltiq (BridgeBio Pharma, Inc.) Formula C26H31Cl2N7O3. H3PO4 …

Ibrexafungerp citrate

It’s only fair to share… Ibrexafungerp citrate アイブレキサフンジェルプクエン酸塩; Formula C44H67N5O4. C6H8O7 cas 1965291-08-0 free 1207753-03-4 Mol weight 922.1574 Brexafemme, fda approved 2021, 2021/6/1 Antifungal, Cell wall biosynthesis inhibitor, Treatment of invasive fungal infections due to Candida spp. or Aspergillus spp., vulvovaginal candidiasis SCY-078 citrate, MK-3118; SCY-078, WHO 10597 UNII-M4NU2SDX3E M4NU2SDX3E (1R,5S,6R,7R,10R,11R,14R,15S,20R,21R)-21-[(2R)-2-amino-2,3,3-trimethylbutoxy]-5,7,10,15-tetramethyl-7-[(2R)-3-methylbutan-2-yl]-20-(5-pyridin-4-yl-1,2,4-triazol-1-yl)-17-oxapentacyclo[13.3.3.01,14.02,11.05,10]henicos-2-ene-6-carboxylic acid;2-hydroxypropane-1,2,3-tricarboxylic acid Originator Merck & Co; SCYNEXIS Class Antifungals; Glycosides; …

Loncastuximab tesirine

It’s only fair to share… Loncastuximab tesirine ZYNLONTA FDA APPROVED 2021/4/23 https://www.adcreview.com/wp-content/uploads/2019/06/15_ICML_Chiara-Tarantelli-ADCT_402_preclinical.pdf Formula C6544H10048N1718O2064S52 Exact mass 147387.9585 CAS 1879918-31-6 Efficacy Antineoplasitc, Anti-CD19 antibody Disease Diffuse large B-cell lymphoma not otherwise specified [DS:H02434] Comment Antibody-drug conjugate Treatment of hematological cancers ロンカスツキシマブテシリン; ADCT-402, ADCX 19 Immunoglobulin G1, anti-(human CD19 antigen) (human-Mus musculus monoclonal RB4v1.2 γ1-chain), disulfide with human-Mus …