It’s only fair to share… Gildeuretinol Retin-20,20,20-d3-ol CAS118139-35-8 MF C20H272H3O, MW 289.5 g/mol (2E,4E,6E,8E)-3-(2H3)methyl-7-methyl-9-(2,6,6-trimethylcyclohex-1-en-1-yl)nona-2,4,6,8-tetraen-1-ol; (20,20,20-2H3)retinol (2E,4E,6E,8E)-7-methyl-3-(trideuteriomethyl)-9-(2,6,6-trimethylcyclohexen-1-yl)nona-2,4,6,8-tetraen-1-olvitamin A analogue, Orphan Drug, Stargardt disease, breakthrough therapy, Pediatric Rare Disease designations, ALK-001, KL-49, ALK 001, KL 49 Gildeuretinol is an investigational new drug being developed by Alkeus Pharmaceuticals, Inc. for the treatment of retinal diseases, particularly Stargardt disease and geographic atrophy secondary to age-related macular degeneration (AMD). Stargardt disease is …
Category «orphan drug»
Claziprotamide
It’s only fair to share… Claziprotamide CAS 2361124-03-8, BBP 671 MF C19H20ClFN4O MW374.8 g/mol 1-[4-(6-chloropyridazin-3-yl)piperazin-1-yl]-2-(4-cyclopropyl-3-fluorophenyl)ethan-1-one 1-[4-(6-chloropyridazin-3-yl)piperazin-1-yl]-2-(4-cyclopropyl-3-fluorophenyl)ethan-1-onepantothenate kinases 1 and 3 (PanK1 and PanK3) positive allosteric modulator Claziprotamide is an investigational new drug that is being evaluated for the treatment of rare metabolic disorders such as pantothenate kinase-associated neurodegeneration (PKAN) and neurodegeneration with brain iron accumulation (NBIA). It acts as a positive allosteric modulator …
Atumelnant
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 …
Asengeprast
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 …
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 …