Tag «Orphan Drug Status»

Pemigatinib

It’s only fair to share…   Pemigatinib INCB054828 Formula C24H27F2N5O4 CAS 1513857-77-6 2379919-96-5  HCL Mol weight 487.4991 2020/4/17FDA APPROVED, PEMAZYRE 佩米替尼 [Chinese] [INN] 3-(2,6-Difluoro-3,5-dimethoxyphenyl)-1-ethyl-8-(morpholinomethyl)-1,3,4,6-tetrahydro-2H-pyrrolo[3′,2′:5,6]pyrido[4,3-d]pyrimidin-2-one 2H-Pyrrolo[3′,2′:5,6]pyrido[4,3-d]pyrimidin-2-one, 3-(2,6-difluoro-3,5-dimethoxyphenyl)-1-ethyl-1,3,4,7-tetrahydro-8-(4-morpholinylmethyl)- 3-(2,6-difluoro-3,5-dimethoxyphenyl)-1-ethyl-8-(morpholin-4-ylmethyl)-1,3,4,7-tetrahydro-2H-pyrrolo[3′,2′:5,6]pyrido[4,3-d]pyrimidin-2-one OriginatorIncyte Corporation DeveloperIncyte Corporation; Innovent Biologics ClassAntineoplastics; Ethers; Fluorobenzenes; Morpholines; Pyridines; Pyrimidinones; Pyrroles; Small molecules Mechanism of ActionType 1 fibroblast growth factor receptor antagonists; Type 3 fibroblast growth factor receptor …

Odevixibat

It’s only fair to share… Odevixibat A-4250, AR-H 064974 CAS 501692-44-0 BUTANOIC ACID, 2-(((2R)-2-((2-((3,3-DIBUTYL-2,3,4,5-TETRAHYDRO-7-(METHYLTHIO)-1,1-DIOXIDO-5-PHENYL-1,2,5-BENZOTHIADIAZEPIN-8-YL)OXY)ACETYL)AMINO)-2-(4-HYDROXYPHENYL)ACETYL)AMINO)-, (2S)- (2S)-2-[[(2R)-2-[[2-[(3,3-dibutyl-7-methylsulfanyl-1,1-dioxo-5-phenyl-2,4-dihydro-1λ6,2,5-benzothiadiazepin-8-yl)oxy]acetyl]amino]-2-(4-hydroxyphenyl)acetyl]amino]butanoic acid Molecular Formula C37H48N4O8S2 Molecular Weight 740.929 AZD8294WHO 10706AR-H064974HY-109120CS-0078340D11716US9694018, 5Originator Albireo AB Developer Albireo AB; Albireo Pharma ClassAcetamides; Butyric acids; Hepatoprotectants; Small molecules; Sulfones; Thiazepines Mechanism of Action Sodium-bile acid cotransporter inhibitors Orphan Drug Status Yes – Primary biliary cirrhosis; Biliary atresia; Intrahepatic cholestasis; Alagille …

TILDACERFONT

It’s only fair to share… TILDACERFONT Synonyms: Tildacerfont 1014983-00-6 3-(4-Chloro-2-morpholin-4-yl-thiazol-5-yl)-7-(1-ethyl-propyl)-2,5-dimethyl-pyrazolo[1,5-a]pyrimidine 7-(1-ethyl-propyl)-3-(4-chloro-2-morpholin-4-yl-thiazol-5-yl)-2,5-dimethyl-pyrazolo[1,5-a]pyrimidine MW/ MF 420 g/mol/ C20H26ClN5OS Originator Spruce Biosciences Class2 ring heterocyclic compounds; Morpholines; Pyrazoles; Pyrimidines; Small molecules; Thiazoles Mechanism of Action Corticotropin receptor antagonists Orphan Drug Status Yes – Congenital adrenal hyperplasia New Molecular Entity Yes Phase II Congenital adrenal hyperplasia 09 Jul 2020 Spruce Biosciences initiates a phase II trial …

Blarcamesine, ブラルカメシン ,

It’s only fair to share… Blarcamesine ブラルカメシン; [(2,2-diphenyloxolan-3-yl)methyl]dimethylamine Anavex 2-73 Tetrahydro-N,N-dimethyl-2,2-diphenyl-3-furanemethanamine THD-DP-FM AE-37 / AE37 / ANAVEX 2-73 FREE BASE UNII 9T210MMZ3F Formula C19H23NO Cas 195615-83-9 195615-84-0 HCL Mol weight 281.392 Treatment of Rett syndrome, Investigated for use/treatment in breast cancer. Anti-amnesic, Muscarinic/sigma receptor agonist Originator Anavex Life Sciences Developer ABX-CRO; Anavex Life Sciences; The Michael J. Fox Foundation for Parkinsons Research Class Antidementias; …

Reldesemtiv

It’s only fair to share… Reldesemtiv CK-2127107 CAS 1345410-31-2 UNII-4S0HBYW6QE, 4S0HBYW6QE MW 384.4 g/mol, MF C19H18F2N6O 1-[2-({[trans-3-fluoro-1-(3-fluoropyridin-2-yl)cyclobutyl]methyl}amino)pyrimidin-5-yl]-1H-pyrrole-3- carboxamide 1-[2-[[3-fluoro-1-(3-fluoropyridin-2-yl)cyclobutyl]methylamino]pyrimidin-5-yl]pyrrole-3-carboxamide Reldesemtiv, also known as CK-2127107, is a skeletal muscle troponin activator (FSTA) and is a potential treatment for people living with debilitating diseases and conditions associated with neuromuscular or non-neuromuscular dysfunction, muscular weakness, and/or muscle fatigue such as SMA, …

MITAPIVAT

It’s only fair to share… MITAPIVAT CAS 1260075-17-9 MF C24H26N4O3S MW 450.55 8-Quinolinesulfonamide, N-[4-[[4-(cyclopropylmethyl)-1-piperazinyl]carbonyl]phenyl]- N-[4-[[4-(Cyclopropylmethyl)-1-piperazinyl]carbonyl]phenyl]-8-quinolinesulfonamide Originator Agios Pharmaceuticals Class Antianaemics; Piperazines; Quinolines; Small molecules; Sulfonamides Mechanism of Action Pyruvate kinase stimulants Orphan Drug Status Yes – Inborn error metabolic disorders New Molecular Entity Yes Phase III Inborn error metabolic disorders Phase II  Thalassaemia 27 Feb 2019 Agios Pharmaceuticals plans a phase III trial for …

Cavosonstat (N-91115)

It’s only fair to share… Cavosonstat (N-91115) CAS 1371587-51-7 C16H10ClNO3, 299.71 g/mol UNII-O2Z8Q22ZE4, O2Z8Q22ZE4, NCT02589236; N91115-2CF-05; SNO-6 3-chloro-4-(6-hydroxyquinolin-2-yl)benzoic acid Treatment of Chronic Obstructive Pulmonary Diseases (COPD), AND Cystic fibrosis,  Nivalis Therapeutics, phase 2 The product was originated at Nivalis Therapeutics, which was acquired by Alpine Immune Sciences in 2017. In 2018, Alpine announced the sale and transfer of global rights to …

Deutivacaftor

It’s only fair to share… Deutivacaftor RN: 1413431-07-8 UNII: SHA6U5FJZL,  N-[2-tert-butyl-4-[1,1,1,3,3,3-hexadeuterio-2-(trideuteriomethyl)propan-2-yl]-5-hydroxyphenyl]-4-oxo-1H-quinoline-3-carboxamide Molecular Formula, C24-H28-N2-O3, Molecular Weight, 401.552 Synonyms CTP-656 D9-ivacaftor Deutivacaftor Ivacaftor D9 UNII-SHA6U5FJZL VX-561 WHO 10704 Treatment of Cystic Fibrosis Originator Concert Pharmaceuticals Class Amides; Aminophenols; Antifibrotics; Organic deuterium compounds; Quinolones; Small molecules Mechanism of Action Cystic fibrosis transmembrane conductance regulator stimulants Orphan Drug Status Yes – Cystic fibrosis Phase II Cystic …

Vatiquinone, バチキノン

It’s only fair to share… Vatiquinone バチキノン Vatiquinone; Alpha-Tocotrienol quinone; EPI-743; UNII-6O85FK9I0X; 1213269-98-7; Vincerenone Molecular Formula: C29H44O3 Molecular Weight: 440.668 g/mol 2-[(3R,6E,10E)-3-hydroxy-3,7,11,15-tetramethylhexadeca-6,10,14-trienyl]-3,5,6-trimethylcyclohexa-2,5-diene-1,4-dione 2-((R,6E,10E)-3-hydroxy-3,7,11,15-tetramethylhexadeca-6,10,14-trien-1-yl)-3,5,6-trimethylcyclohexa-2,5-diene-1,4-dione 2-[(3R,6E,10E)-3-hydroxy-3,7,11,15-tetramethylhexadeca-6,10,14-trien-1-yl]-3,5,6-trimethylcyclohexa-2,5-diene-1,4-dione 6O85FK9I0X 9604 Research Code:EPI-743; ATQ-3, BioE-743 MOA:Mitochondria Originator Edison Pharmaceuticals Developer Edison Pharmaceuticals; Sumitomo Dainippon Pharma; University of Florida; Yale University Class Alkadienes; Benzoquinones; Cyclohexenes; Small molecules Mechanism of Action Antioxidants; NQO1 modulators Orphan Drug Status Yes – Mitochondrial …

Glasdegib, PF-04449913

It’s only fair to share…   Glasdegib (PF-04449913) 1-[(2R,4R)-2-(1H-Benzimidazol-2-yl)-1-methyl-4-piperidinyl]-3-(4-cyanophenyl)urea [ACD/IUPAC Name] 1-[(2R,4R)-2-(1H-benzimidazol-2-yl)-1-methylpiperidin-4-yl]-3-(4-cyanophenyl)urea CAS 1095173-27-5 [RN], Orphan Drug Status Glasdegib Molecular FormulaC21H22N6O Average mass374.439 Da Urea, N-[(2R,4R)-2-(1H-benzimidazol-2-yl)-1-methyl-4-piperidinyl]-N’-(4-cyanophenyl)- [ACD/Index Name] гласдегиб [Russian] [INN] غلاسديغيب [Arabic] [INN] 格拉德吉 [Chinese] [INN] FACT SHEET   https://www.pfizer.com/files/news/asco/Glasdegib-Fact-Sheet-6JUNE2018.pdf Glasdegib (PF-04449913) is an experimental cancer drug developed by Pfizer. It is a small molecule inhibitor of the Sonic hedgehog pathway, which is overexpressed in many types of …