Category «Orphan Drug Status»

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 …

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; …

PACRITINIB

It’s only fair to share…   Pacritinib パクリチニブ; Formula C28H32N4O3 CAS 937272-79-2 Mol weight 472.5787 UPDATE  FDA APPROVED  2/28/2022, Vonjo To treat intermediate or high-risk primary or secondary myelofibrosis in adults with low platelets A Jak2 inhibitor potentially for the treatment of acute myeloid Leukemia and myelofibrosis. UNII-G22N65IL3O пакритиниб باكريتينيب 帕瑞替尼 ONX-0803; SB-1518 CAS No. …

Efgartigimod alfa-fcab

It’s only fair to share… DKTHTCPPCP APELLGGPSV FLFPPKPKDT LYITREPEVT CVVVDVSHED PEVKFNWYVD GVEVHNAKTK PREEQYNSTY RVVSVLTVLH QDWLNGKEYK CKVSNKALPA PIEKTISKAK GQPREPQVYT LPPSRDELTK NQVSLTCLVK GFYPSDIAVE WESNGQPENN YKTTPPVLDS DGSFFLYSKL TVDKSRWQQG NVFSCSVMHE ALKFHYTQKS LSLSPGK (Disulfide bridge: 6-6′, 9-9′, 41-101, 147-205, 41′-101′, 147′-205′) Efgartigimod alfa-fcab Formula C2310H3554N602O692S14 CAS 1821402-21-4 Mol weight 51279.464 US FDA APPROVED 12/17/2021, To treat generalized myasthenia gravis Press Release, Vyvgart,  BLA 761195 …