Tag «Orphan Drug Status»

It’s only fair to share… Atumelnant CAS 2392970-97-5 MF C33H42F3N5O3 MW 613.7 g/mol CRN04894, NR57FH6U1N CRINETICS PHARMA, Orphan Drug Status, Congenital adrenal hyperplasia N-[(3S)-1-azabicyclo[2.2.2]octan-3-yl]-6-(2-ethoxyphenyl)-3-[(2R)-2-ethyl-4-[1-(trifluoromethyl)cyclobutanecarbonyl]piperazin-1-yl]pyridine-2-carboxamide N-[(3S)-1-azabicyclo[2.2.2]octan-3-yl]-6-(2-ethoxyphenyl)-3-{(2R)-2-ethyl-4-[1-(trifluoromethyl) cyclobutane-1-carbonyl]piperazin-1-yl}pyridine-2-carboxamideAdrenocorticotropic hormone receptor antagonist Atumelnant (INNTooltip International Nonproprietary Name; developmental code name CRN04894) is an investigational new drug developed by Crinetics Pharmaceuticals for the treatment of adrenocorticotropic hormone (ACTH)-dependent endocrine disorders.[1] It is a selective antagonist of the melanocortin type 2 receptor (MC2R), also …

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It’s only fair to share… Asengeprast CAS 1001288-58-9 FT011, FT 011, orphan drug status, systemic sclerosis, SHP-627, SHP 627, Fast Track 2-[[(E)-3-(3-methoxy-4-prop-2-ynoxyphenyl)prop-2-enoyl]amino]benzoic acid 2-[(2E)-3-{3-methoxy-4-[(prop-2-yn-1-yl)oxy]phenyl}prop-2-enamido]benzoic acid G protein-coupled receptor 68 (GPR68) antagonist,anti-inflammatory MF C20H17NO5 MW 351.4 g/mol. C6V7ZU2NPR Asengeprast (development code FT011) is an experimental scleroderma drug candidate.[1] It is a small molecule inhibitor of the G-protein coupled receptor GPR68 with antifibrotic activity.[2] It is …

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It’s only fair to share… (Heavy chain) QVQLVQSGAE VKKPGASVKV SCKASGYSFT SSWIHWVKQA PGQGLEWMGE INPGNVRTNY NENFRNKVTM TVDTSISTAY MELSRLRSDD TAVYYCTVVF YGEPYFPYWG QGTLVTVSSA STKGPSVFPL APSSKSTSGG TAALGCLVKD YFPEPVTVSW NSGALTSGVH TFPAVLQSSG LYSLSSVVTV PSSSLGTQTY ICNVNHKPSN TKVDKRVEPK SCDKTHTCPP CPAPEAAGGP SVFLFPPKPK DTLMISRTPE VTCVVVDVSH EDPEVKFNWY VDGVEVHNAK TKPREEQYNS TYRVVSVLTV LHQDWLNGKE YKCKVSNKAL PAPIEKTISK AKGQPREPQV YTLPPSREEM TKNQVSLTCL VKGFYPSDIA VEWESNGQPE NNYKTTPPVL DSDGSFFLYS KLTVDKSRWQ QGNVFSCSVM HEALHNHYTQ KSLSLSPGK (Light chain) QIVLTQSPGT …

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It’s only fair to share… MIRDAMETINIB 391210-10-9 Chemical Formula: C16H14F3IN2O4 Molecular Weight: 482.19 PD0325901; PD 0325901; PD-325901; mirdametinib FDA APPROVED  2/11/2025, Gomekli, To treat neurofibromatosis type 1 who have symptomatic plexiform neurofibromas not amenable to complete resection 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 …

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It’s only fair to share…       Dasiglucagon Treatment of Hypoglycemia in Type 1 and Type 2 Diabetes Patients Formula C152H222N38O50 CAS 1544300-84-6 Mol weight 3381.6137 FDA APPROVED,  2021/3/22, Zegalogue Zealand Pharma A/S UNIIAD4J2O47FQ HypoPal rescue pen SVG Image IUPAC Condensed H-His-Ser-Gln-Gly-Thr-Phe-Thr-Ser-Asp-Tyr-Ser-Lys-Tyr-Leu-Asp-Aib-Ala-Arg-Ala-Glu-Glu-Phe-Val-Lys-Trp-Leu-Glu-Ser-Thr-OH Sequence HSQGTFTSDYSKYLDXARAEEFVKWLEST HELM PEPTIDE1{H.S.Q.G.T.F.T.S.D.Y.S.K.Y.L.D.[Aib].A.R.A.E.E.F.V.K.W.L.E.S.T}$$$$ IUPAC L-histidyl-L-seryl-L-glutaminyl-glycyl-L-threonyl-L-phenylalanyl-L-threonyl-L-seryl-L-alpha-aspartyl-L-tyrosyl-L-seryl-L-lysyl-L-tyrosyl-L-leucyl-L-alpha-aspartyl-alpha-methyl-alanyl-L-alanyl-L-arginyl-L-alanyl-L-alpha-glutamyl-L-alpha-glutamyl-L-phenylalanyl-L-valyl-L-lysyl-L-tryptophyl-L-leucyl-L-alpha-glutamyl-L-seryl-L-threonine (4S)-4-[[(2S)-2-[[(2S)-2-[[(2S)-2-[[2-[[(2S)-2-[[(2S)-2-[[(2S)-2-[[(2S)-6-amino-2-[[(2S)-2-[[(2S)-2-[[(2S)-2-[[(2S)-2-[[(2S,3R)-2-[[(2S)-2-[[(2S,3R)-2-[[2-[[(2S)-5-amino-2-[[(2S)-2-[[(2S)-2-amino-3-(1H-imidazol-4-yl)propanoyl]amino]-3-hydroxypropanoyl]amino]-5-oxopentanoyl]amino]acetyl]amino]-3-hydroxybutanoyl]amino]-3-phenylpropanoyl]amino]-3-hydroxybutanoyl]amino]-3-hydroxypropanoyl]amino]-3-carboxypropanoyl]amino]-3-(4-hydroxyphenyl)propanoyl]amino]-3-hydroxypropanoyl]amino]hexanoyl]amino]-3-(4-hydroxyphenyl)propanoyl]amino]-4-methylpentanoyl]amino]-3-carboxypropanoyl]amino]-2-methylpropanoyl]amino]propanoyl]amino]-5-carbamimidamidopentanoyl]amino]propanoyl]amino]-5-[[(2S)-1-[[(2S)-1-[[(2S)-1-[[(2S)-6-amino-1-[[(2S)-1-[[(2S)-1-[[(2S)-4-carboxy-1-[[(2S)-1-[[(1S,2R)-1-carboxy-2-hydroxypropyl]amino]-3-hydroxy-1-oxopropan-2-yl]amino]-1-oxobutan-2-yl]amino]-4-methyl-1-oxopentan-2-yl]amino]-3-(1H-indol-3-yl)-1-oxopropan-2-yl]amino]-1-oxohexan-2-yl]amino]-3-methyl-1-oxobutan-2-yl]amino]-1-oxo-3-phenylpropan-2-yl]amino]-4-carboxy-1-oxobutan-2-yl]amino]-5-oxopentanoic acid . [16-(2-methylalanine)(S>X),17-L-alanine(R>A),20-L-α-glutamyl(Q>E),21-L-αglutamyl(D>E),24-L-lysyl(Q>K),27-L-α-glutamyl(M>E),28-L-serine(N>S)]human glucagon L-Threonine, L-histidyl-L-seryl-L-glutaminylglycyl-L-threonyl-L- …

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It’s only fair to share…   Pemigatinib INCB054828 Formula C24H27F2N5O4 CAS 1513857-77-6 2379919-96-5  HCL Mol weight 487.4991 2020/4/17FDA APPROVED, PEMAZYRE 佩米替尼 [Chinese] [INN] 3-(2,6-Difluoro-3,5-dimethoxyphenyl)-1-ethyl-8-(morpholinomethyl)-1,3,4,6-tetrahydro-2H-pyrrolo[3′,2′:5,6]pyrido[4,3-d]pyrimidin-2-one 2H-Pyrrolo[3′,2′:5,6]pyrido[4,3-d]pyrimidin-2-one, 3-(2,6-difluoro-3,5-dimethoxyphenyl)-1-ethyl-1,3,4,7-tetrahydro-8-(4-morpholinylmethyl)- 3-(2,6-difluoro-3,5-dimethoxyphenyl)-1-ethyl-8-(morpholin-4-ylmethyl)-1,3,4,7-tetrahydro-2H-pyrrolo[3′,2′:5,6]pyrido[4,3-d]pyrimidin-2-one OriginatorIncyte Corporation DeveloperIncyte Corporation; Innovent Biologics ClassAntineoplastics; Ethers; Fluorobenzenes; Morpholines; Pyridines; Pyrimidinones; Pyrroles; Small molecules Mechanism of ActionType 1 fibroblast growth factor receptor antagonists; Type 3 fibroblast growth factor receptor …

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It’s only fair to share… Odevixibat A-4250, AR-H 064974 CAS 501692-44-0 BUTANOIC ACID, 2-(((2R)-2-((2-((3,3-DIBUTYL-2,3,4,5-TETRAHYDRO-7-(METHYLTHIO)-1,1-DIOXIDO-5-PHENYL-1,2,5-BENZOTHIADIAZEPIN-8-YL)OXY)ACETYL)AMINO)-2-(4-HYDROXYPHENYL)ACETYL)AMINO)-, (2S)- (2S)-2-[[(2R)-2-[[2-[(3,3-dibutyl-7-methylsulfanyl-1,1-dioxo-5-phenyl-2,4-dihydro-1λ6,2,5-benzothiadiazepin-8-yl)oxy]acetyl]amino]-2-(4-hydroxyphenyl)acetyl]amino]butanoic acid Molecular Formula C37H48N4O8S2 Molecular Weight 740.929 AZD8294WHO 10706AR-H064974HY-109120CS-0078340D11716US9694018, 5Originator Albireo AB Developer Albireo AB; Albireo Pharma ClassAcetamides; Butyric acids; Hepatoprotectants; Small molecules; Sulfones; Thiazepines Mechanism of Action Sodium-bile acid cotransporter inhibitors Orphan Drug Status Yes – Primary biliary cirrhosis; Biliary atresia; Intrahepatic cholestasis; Alagille …

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It’s only fair to share… TILDACERFONT Synonyms: Tildacerfont 1014983-00-6 3-(4-Chloro-2-morpholin-4-yl-thiazol-5-yl)-7-(1-ethyl-propyl)-2,5-dimethyl-pyrazolo[1,5-a]pyrimidine 7-(1-ethyl-propyl)-3-(4-chloro-2-morpholin-4-yl-thiazol-5-yl)-2,5-dimethyl-pyrazolo[1,5-a]pyrimidine MW/ MF 420 g/mol/ C20H26ClN5OS Originator Spruce Biosciences Class2 ring heterocyclic compounds; Morpholines; Pyrazoles; Pyrimidines; Small molecules; Thiazoles Mechanism of Action Corticotropin receptor antagonists Orphan Drug Status Yes – Congenital adrenal hyperplasia New Molecular Entity Yes Phase II Congenital adrenal hyperplasia 09 Jul 2020 Spruce Biosciences initiates a phase II trial …

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It’s only fair to share… Blarcamesine ブラルカメシン; [(2,2-diphenyloxolan-3-yl)methyl]dimethylamine Anavex 2-73 Tetrahydro-N,N-dimethyl-2,2-diphenyl-3-furanemethanamine THD-DP-FM AE-37 / AE37 / ANAVEX 2-73 FREE BASE UNII 9T210MMZ3F Formula C19H23NO Cas 195615-83-9 195615-84-0 HCL Mol weight 281.392 Treatment of Rett syndrome, Investigated for use/treatment in breast cancer. Anti-amnesic, Muscarinic/sigma receptor agonist Originator Anavex Life Sciences Developer ABX-CRO; Anavex Life Sciences; The Michael J. Fox Foundation for Parkinsons Research Class Antidementias; …

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It’s only fair to share… Reldesemtiv CK-2127107 CAS 1345410-31-2 UNII-4S0HBYW6QE, 4S0HBYW6QE MW 384.4 g/mol, MF C19H18F2N6O 1-[2-({[trans-3-fluoro-1-(3-fluoropyridin-2-yl)cyclobutyl]methyl}amino)pyrimidin-5-yl]-1H-pyrrole-3- carboxamide 1-[2-[[3-fluoro-1-(3-fluoropyridin-2-yl)cyclobutyl]methylamino]pyrimidin-5-yl]pyrrole-3-carboxamide Reldesemtiv, also known as CK-2127107, is a skeletal muscle troponin activator (FSTA) and is a potential treatment for people living with debilitating diseases and conditions associated with neuromuscular or non-neuromuscular dysfunction, muscular weakness, and/or muscle fatigue such as SMA, …

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