Category «Orphan Drug Status»

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 …

MIRDAMETINIB

It’s only fair to share… MIRDAMETINIB 391210-10-9 Chemical Formula: C16H14F3IN2O4 Molecular Weight: 482.19 PD0325901; PD 0325901; PD-325901; mirdametinib IUPAC/Chemical Name: (R)-N-(2,3-dihydroxypropoxy)-3,4-difluoro-2-((2-fluoro-4-iodophenyl)amino)benzamide SpringWorks Therapeutics (a spin out of Pfizer ) is developing mirdametinib, a second-generation, non-ATP competitive, allosteric MEK1 and MEK2 inhibitor derived from CI-1040, for treating type 1 neurofibromatosis (NF1) and advanced solid tumors. In June …

BELUMOSUDIL

It’s only fair to share… BELUMOSUDIL C26H24N6O2 MW 452.5 911417-87-3, SLx-2119, KD-025, KD 025, WHO 11343 2-[3-[4-(1H-indazol-5-ylamino)quinazolin-2-yl]phenoxy]-N-propan-2-ylacetamide 2-(3-(4-(lH-indazol-5-ylamino)quinazolin-2-yl)phenoxy)-N-isopropylacetamide Belumosudil mesylate KD025 mesylate 2109704-99-4 Belumosudil mesylate is an orally available rho kinase 2 (ROCK 2) inhibitor being developed at Kadmon. In 2020, the drug candidate was submitted for a new drug application (NDA) in the U.S., under a …

Asparaginase erwinia chrysanthemi (recombinant)-rywn

It’s only fair to share… Sequence: 1ADKLPNIVIL ATGGTIAGSA ATGTQTTGYK AGALGVDTLI NAVPEVKKLA 51NVKGEQFSNM ASENMTGDVV LKLSQRVNEL LARDDVDGVV ITHGTDTVEE 101SAYFLHLTVK SDKPVVFVAA MRPATAISAD GPMNLLEAVR VAGDKQSRGR 151GVMVVLNDRI GSARYITKTN ASTLDTFKAN EEGYLGVIIG NRIYYQNRID 201KLHTTRSVFD VRGLTSLPKV DILYGYQDDP EYLYDAAIQH GVKGIVYAGM 251GAGSVSVRGI AGMRKAMEKG VVVIRSTRTG NGIVPPDEEL PGLVSDSLNP 301AHARILLMLA LTRTSDPKVI QEYFHTY >Protein sequence for asparaginase (Erwinia chrysanthemi) monomer ADKLPNIVILATGGTIAGSAATGTQTTGYKAGALGVDTLINAVPEVKKLANVKGEQFSNM ASENMTGDVVLKLSQRVNELLARDDVDGVVITHGTDTVEESAYFLHLTVKSDKPVVFVAA MRPATAISADGPMNLLEAVRVAGDKQSRGRGVMVVLNDRIGSARYITKTNASTLDTFKAN EEGYLGVIIGNRIYYQNRIDKLHTTRSVFDVRGLTSLPKVDILYGYQDDPEYLYDAAIQH GVKGIVYAGMGAGSVSVRGIAGMRKAMEKGVVVIRSTRTGNGIVPPDEELPGLVSDSLNP AHARILLMLALTRTSDPKVIQEYFHTY References: Therapeutic Targets …

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 …

Pegcetacoplan

It’s only fair to share… Sequence: 1ICVWQDWGAH RCTXK Sequence: 1ICVWQDWGAH RCTXK Sequence Modifications Type Location Description terminal mod. Lys-15 C-terminal amide terminal mod. Lys-15′ C-terminal amide bridge Cys-2 – Cys-12 disulfide bridge, dimer bridge Lys-15 – Lys-15′ covalent bridge, dimer bridge Cys-2′ – Cys-12′ disulfide bridge, dimer uncommon Oaa-14 – uncommon Oaa-14′ – Pegcetacoplan ペグセタコプラン; …

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 …