Category «orphan drug»

HEXASODIUM PHYTATE

It’s only fair to share… HEXASODIUM PHYTATE cas 34367-89-0 myo-Inositol, 1,2,3,4,5,6-hexakis(dihydrogen phosphate) sodium salt (1:6) hexasodium;[2,3,4,5,6-pentakis[[hydroxy(oxido)phosphoryl]oxy]cyclohexyl] hydrogen phosphate free form RN: 83-86-3 C6H12Na6O24P6, 791.93 x Na salt 14306-25-3 C6H18O24P6.xNa free form : 83-86-3 myo-Inositol, 1,2,3,4,5,6-hexakis(dihydrogen phosphate), sodium salt (1R,2R,3S,4S,5R,6S)-CYCLOHEXANE-1,2,3,4,5,6-HEXAYL-HEXAKIS(DIHYDROGEN PHOSPHATE) INOSITOL, HEXAKIS(DIHYDROGEN PHOSPHATE) HEXASODIUM SALT, MYO- Hexasodium phytate (also known as SNF472) is a compound being developed as …

Clofutriben

It’s only fair to share… Clofutriben Cas 1204178-50-6 HCL 1203941-88-1 Molecular Weight 424.80 Formula C19H16ClF3N4O2 Clofutriben (ASP3662) is a 11β-hydroxysteroid dehydrogenase type 1 inhibitor. Clofutriben is an orally bioavailable selective inhibitor of the enzyme 11beta-hydroxysteroid dehydrogenase type 1 (11b-HSD1; 11bHSD1; HSD11B1; HSD1; HSD-1), with potential protective activity for disorders of corticosteroid excess. Upon oral administration, …

Levacetylleucine

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 …

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 …

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; …