Category «orphan drug»

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; …

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 …

ZY 19489, MMV 253

It’s only fair to share… ZY 19489, MMV 253 C24 H32 FN9, 465.5 CAS 1821293-40-6 MMV253, GTPL10024, MMV674253 N-(4-cyclopropyl-5-fluoro-6-methylpyridin-2-yl)-5-((3R)-2-((1,5-dimethyl-1H-pyrazol-3-yl)amino)-3,4-dimethylpiperazin-1-yl)pyrimidin-2-amine 2-N-(4-cyclopropyl-5-fluoro-6-methylpyridin-2-yl)-5-[(3R)-3,4-dimethylpiperazin-1-yl]-4-N-(1,5-dimethylpyrazol-3-yl)pyrimidine-2,4-diamine N2-(4-Cyclopropyl-5-fluoro-6-methyl-2-pyridinyl)-5-[(3R)-3,4-dimethyl-1-piperazinyl]-N4-(1,5-dimethyl-1H-pyrazol-3-yl)-2,4-pyrimidinediamine (R)-N2-(4-Cyclopropyl-5-fluoro-6-methylpyridin-2-yl)-N4-(1,5-dimethyl-1H-pyrazol-3-yl)-5-(3,4-dimethylpiperazin-1-yl)pyrimidine-2,4-diamine SYN IN 201721031453 The invention relates to triaminopyrimidine compd. of formula I, pharmaceutically acceptable salts thereof, hydrates, solvates, polymorphs, optically active forms thereof, in solid state forms useful for preventing or treating malaria.  The invention also relates …

Maribavir

It’s only fair to share…   Maribavir Molecular FormulaC15H19Cl2N3O4 Average mass376.235 Da FDA APROVED 11/23/2021, Livtencity 1263 W94, 1263W94 176161-24-3 [RN] 1H-Benzimidazol-2-amine, 5,6-dichloro-N-(1-methylethyl)-1-β-L-ribofuranosyl- UNII-PTB4X93HE1, марибавир , ماريبافير  ,马立巴韦 , BW-1263W94 Camvia, D04859, G1263, GW257406X 1263W94; BW-1263W94; GW-1263; GW-257406X; SHP-620; VP-41263 Company:GlaxoSmithKline (Originator) , Shire MOA:UL97 kinase inhibitor Indication:CMV prophylaxis To treat post-transplant cytomegalovirus (CMV) infection/disease that does not respond (with or without genetic mutations that cause resistance) to available antiviral …