It’s only fair to share… Levacetylleucine WeightAverage: 173.212Monoisotopic: 173.105193347 Chemical FormulaC8H15NO3 (2S)-2-acetamido-4-methylpentanoic acid FDA APPROVED 9/24/2024, To treat Niemann-Pick disease type CPress ReleaseDrug Trials Snapshot Registered Niemann-Pick disease type C 18 Mar 2025Phase-III clinical trials in Ataxia telangiectasia (In adolescents, In children, In the elderly, In adults) in Switzerland, Slovakia, Spain, Germany, USA, United Kingdom …
Tag «orphan drug»
Crinecerfont
It’s only fair to share… Crinecerfont CAS 752253-39-7 SSR125543 SSR 125543 SSR-125543, WHO 10958, UNII-MFT24BX55I, 06-RORI,NBI-74788 FDA APPROVED 12/13/2024, Crenessity, To treat classic congenital adrenal hyperplasia Press Release 4-(2-chloro-4-methoxy-5-methylphenyl)-N-[(1S)-2-cyclopropyl-1-(3-fluoro-4-methylphenyl)ethyl]-5-methyl-N-prop-2-ynyl-1,3-thiazol-2-amine WeightAverage: 483.04Monoisotopic: 482.1594906 Chemical FormulaC27H28ClFN2OS CAS No. : 321839-75-2 Molecular Weight 519.50 Formula C27H29Cl2FN2OS Crinecerfont, sold under the brand name Crenessity, is a medication used for the treatment of congenital adrenal hyperplasia.[1] It …
Relacorilant
It’s only fair to share… Relacorilant Molecular FormulaC27H22F4N6O3S Average mass586.561 Da CAS 1496510-51-0 Phase III UNII-2158753C7E 2158753C7E [(4aR)-1-(4-fluorophenyl)-6-(1-methylpyrazol-4-yl)sulfonyl-4,5,7,8-tetrahydropyrazolo[3,4-g]isoquinolin-4a-yl]-[4-(trifluoromethyl)pyridin-2-yl]methanone [(4aR)-1-(4-fluorophenyl)-6-(1-methylpyrazol-4-yl)sulfonyl-4,5,7,8-tetrahydropyrazolo[3,4-g]isoquinolin-4a-yl]-[4-(trifluoromethyl)pyridin-2-yl]methanone Methanone, [(4aR)-1-(4-fluorophenyl)-1,4,5,6,7,8-hexahydro-6-[(1-methyl-1H-pyrazol-4-yl)sulfonyl]-4aH-pyrazolo[3,4-g]isoquinolin-4a-yl][4-(trifluoromethyl)-2-pyridinyl]- Methanone, ((4aR)-1-(4-fluorophenyl)-1,4,5,6,7,8-hexahydro-6-((1-methyl-1H-pyrazol-4-yl)sulfonyl)-4ah-pyrazolo(3,4-g)isoquinolin-4a-yl)(4-(trifluoromethyl)-2-pyridinyl)- релакорилант [Russian] [INN] ريلاكوريلانت [Arabic] [INN] 瑞拉可兰 [Chinese] [INN] OriginatorCorcept Therapeutics ClassAntineoplastics; Fluorine compounds; Isoquinolines; Ketones; Organic sulfur compounds; Pyrazoles; Pyridines; Small molecules Mechanism of ActionGlucocorticoid receptor antagonists Orphan Drug StatusYes – Pancreatic cancer; Cushing syndrome Phase …
Milademetan
It’s only fair to share… Milademetan Molecular Weight 618.53 Formula C30H34Cl2FN5O4 CAS No. 1398568-47-2 Milademetan. hcl Chemical Formula: C30H35Cl3FN5O4 Exact Mass: 617.1972 Molecular Weight: 654.99 Elemental Analysis: C, 55.01; H, 5.39; Cl, 16.24; F, 2.90; N, 10.69; O, 9.77 1398568-47-2 (free base) 1398569-75-9 (tosylate) 2095625-97-9 (tosylate hydrate) Milademetan HCl DS3032b; DS-3032b; DS 3032b; DS3032; …
Daxibotulinumtoxin A
It’s only fair to share… Daxibotulinumtoxin A FDA APPROVED 2022 2022/9/7, Daxxify Formula C6708H10359N1729O1995S32 CAS 93384-43-1 Mol weight 148171.4934 Daxibotulinumtoxin A-lanm Treatment of galbellar lines, cervical dystonia, lateral canthal lines, migraine headaches and hyperhidrosis DeveloperRevance Therapeutics; Shanghai Fosun Pharmaceutical ClassAnalgesics; Anti-inflammatories; Antiarrhythmics; Antidepressants; Antimigraines; Antipruritics; Antispasmodics; Bacterial proteins; Bacterial toxins; Botulinum toxins; Eye disorder therapies; Foot …
ELRAGLUSIB
It’s only fair to share… ELRAGLUSIB RN: 1034895-42-5 UNII: ND1SOF0DLU, WHO 11553, 9-ING-41 1H-Pyrrole-2,5-dione, 3-(5-fluoro-3-benzofuranyl)-4-(5-methyl-5H-1,3-dioxolo(4,5-F)indol-7-yl)- 3-(5-Fluoro-benzofuran-3-yl)-4-(5-methyl-5H-(1,3)dioxolo(4,5-F)indol-7-yl)-pyrrole-2,5-dione Antineoplastic Molecular Formula C22-H13-F-N2-O5 Molecular Weight 404.3517 OriginatorNorthwestern University; University of Illinois at Chicago DeveloperActuate Therapeutics; Incyte Corporation; Levine Cancer Institute; University of Kansas Medical Center ClassAntineoplastics; Benzofurans; Dioxolanes; Indoles; Pyrroles; Small molecules Mechanism of ActionGlycogen synthase kinase 3 beta inhibitors Orphan …
Valemetostat tosilate
It’s only fair to share… Valemetostat tosilate バレメトスタットトシル酸塩 Formula C26H34ClN3O4. C7H8O3S CAS 1809336-93-3 Mol weight 660.2205 PMDA JAPAN approved 2022/9/26, Ezharmia 1,3-Benzodioxole-5-carboxamide, 7-chloro-N-((1,2-dihydro-4,6-dimethyl-2-oxo-3-pyridinyl)methyl)-2-(trans-4-(dimethylamino)cyclohexyl)-2,4-dimethyl-, (2R)-, compd. with 4-methylbenzenesulfonate (1:1) Antineoplastic, histone methyltransferase inhibitor 1809336-39-7 (free base). 1809336-93-3 (tosylate) 1809336-92-2 (mesylate) 1809336-94-4 (fumarate) 1809336-95-5 (tarate) Synonym: Valemetostat; DS-3201; DS 3201; DS3201; DS-3201b (2R)-7-Chloro-2-[trans-4-(dimethylamino)cyclohexyl]-N-[(4,6-dimethyl-2-oxo-1,2-dihydropyridin-3-yl)methyl]-2,4-dimethyl-1,3-benzodioxole-5-carboxamide mono(4-methylbenzenesulfonate) C26H34ClN3O4▪C7H8O3S : 660.22 …
Danavorexton, TAK 925
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 …
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 …