Tag «orphan drug»

Sparsentan

It’s only fair to share…   Sparsentan (PS433540,RE-021) C32H40N4O5S Average mass592.749 FDA APPROVED 2023/2/17, Filspari 4′-((2-butyl-4-oxo-1,3-diazaspiro[4.4]non-1-en-3-yl)methyl)-N-(4,5-dimethylisoxazol-3-yl)-2′-(ethoxymethyl)-[1,1′-biphenyl]-2-sulfonamide  4′-[(2-Butyl-4-oxo-1.3-diazaspiro[4.41non-l-en-3-yl)methvn-N-(3,4- dimethyl-5-isoxazolyl)-2′-ethoxymethyl [ 1 , l’-biphenyll -2-sulfonamide SparsentanPS433540; RE-021, formerly known as DARACAS :254740-64-24-[(2-butyl-4-oxo-1,3-diazaspiro[4.4]non-1-en-3-yl)methyl]-N-(4,5- dimethylisoxazol-3-yl)-2-(ethoxymethyl)biphenyl-2-sulfonamideMechanism of Action:acting as both an Endothelin Receptor Antagonist (ERA) and Angiotensin Receptor Blocker (ARB).Indication: Focal Segmental Glomerulosclerosis (FSGS).Focal Segmental Glomerulosclerosis (FSGS) is a rare …

Tebapivat

It’s only fair to share… Tebapivat CAS 2283422-04-6 WeightAverage: 392.44Monoisotopic: 392.116778341 Chemical FormulaC18H16N8OS 10-[(6-aminopyridin-2-yl)methyl]-7-methyl-4-(1H-pyrazol-5-ylmethyl)-3-thia-5,7,10,11-tetrazatricyclo[6.4.0.02,6]dodeca-1(8),2(6),4,11-tetraen-9-one6-[(6-aminopyridin-2-yl)methyl]-4-methyl-2-[(1H-pyrazol-3-yl)methyl]-4,6-dihydro-5H-[1,3]thiazolo[5′,4′:4,5]pyrrolo[2,3-d]pyridazin-5-one 6-[(6-aminopyridin-2-yl)methyl]-4-methyl-2-[(1H-pyrazol-3-yl)methyl]-4,6-dihydro-5H-[1,3]thiazolo[5′,4′:4,5]pyrrolo[2,3-d]pyridazin-5-one Tebapivat is under investigation in clinical trial NCT05490446 (A Study of Tebapivat (AG-946) in Participants With Anemia Due to Lower-risk Myelodysplastic Syndromes (LR-MDS)). Tebapivat is an orally available activator of the red cell isoform of pyruvate kinase (PK-R; PKR), with potential to …

PALTUSOTINE

It’s only fair to share… PALTUSOTINE CAS 2172870-89-0 WeightAverage: 456.497Monoisotopic: 456.17616767 Chemical FormulaC27H22F2N4O 3-[4-(4-Amino-1-piperidinyl)-3-(3,5-difluorophenyl)-6-quinolinyl]-2-hydroxybenzonitrile Paltusotine is a selective somatostatin receptor type 2 (SST2) agonist in development by Crinetics Pharmaceuticals for the treatment of acromegaly and certain neuroendocrine tumors. It is a small molecule delivered orally.[1][2][3][4] SCHEME PAPER https://pubs.acs.org/doi/10.1021/acsmedchemlett.2c00431 Discovery of Paltusotine (CRN00808), a Potent, Selective, and Orally Bioavailable Non-peptide SST2 Agonist Step …

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 …

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 …