Tag «Orphan Drug Status»

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 …

Copanlisib

It’s only fair to share… Copanlisib, BAY 80-6946,  BAY 84-1236 Molecular FormulaC23H28N8O4 Average mass480.520 Da Cas 1032568-63-0 [RN] 1402152-26-4 MONO HCL UNII-WI6V529FZ9 FDA Approved September 2017 2-Amino-N-{7-methoxy-8-[3-(4-morpholinyl)propoxy]-2,3-dihydroimidazo[1,2-c]quinazolin-5-yl}-5-pyrimidinecarboxamide 5-Pyrimidinecarboxamide, 2-amino-N-[2,3-dihydro-7-methoxy-8-[3-(4-morpholinyl)propoxy]imidazo[1,2-c]quinazolin-5-yl]- orphan drug status for follicular lymphoma Copanlisib (BAY 80-6946), developed by Bayer, is a selective Class I phosphoinositide 3-kinase inhibitor[1] which has shown promise in Phase I/II clinical trials for the treatment of non-Hodgkin lymphoma and chronic lymphocytic leukemia.[2] …

Funapide, TV 45070, XEN-402, фунапид فونابيد 呋纳匹特

It’s only fair to share… Funapide TV 45070,  XEN-402,  Funapide, (+)- фунапид فونابيد 呋纳匹特 Molecular FormulaC22H14F3NO5 Average mass429.345 Da (S)-1′-[(5-Methyl-2-furyl)methyl]spiro[6H-furo[3,2-f][1,3]benzodioxole-7,3′-indoline]-2′-one Spiro(furo(2,3-F)-1,3-benzodioxole-7(6H),3′-(3H)indol)-2′(1’H)-one, 1′-((5-(trifluoromethyl)-2-furanyl)methyl)-, (3’S)- (3’S)-1′-((5-(Trifluoromethyl)furan-2-yl)methyl)-2H,6H-spiro(furo(2,3-F)(1,3)benzodioxole-7,3′-indol)-2′(1’H)-one Spiro[furo[2,3-f]-1,3-benzodioxole-7(6H),3′-[3H]indol]-2′(1’H)-one, 1′-[[5-(trifluoromethyl)-2-furanyl]methyl]-, (7S)- TV-45070 UNII-A5595LHJ2L XEN-401-S XEN402 (3’S)-1′-{[5-(trifluoromethyl)furan-2-yl]methyl}-2H-6H-spiro[furo[2,3-f]-1,3-benzodioxole-7,3′-indol]-2′(1’H)-one (7S)-1′-{[5-(Trifluoromethyl)-2-furyl]methyl}spiro[furo[2,3-f][1,3]benzodioxole-7,3′-indol]-2′(1’H)-one 1259933-16-8 CAS UNII-A5595LHJ2L Phase II clinical trials for Postherpetic neuralgia (PHN) Treatment of Neuropathic Pain Originator Xenon Pharmaceuticals Developer Teva Pharmaceutical Industries; Xenon Pharmaceuticals Class Benzodioxoles; Fluorobenzenes; …

Enasidenib, Энасидениб , إيناسيدينيب ,伊那尼布 ,

It’s only fair to share…   Enasidenib Molecular Formula C19H17F6N7O Average mass 473.375 2-Propanol, 2-methyl-1-[[4-[6-(trifluoromethyl)-2-pyridinyl]-6-[[2-(trifluoromethyl)-4-pyridinyl]amino]-1,3,5-triazin-2-yl]amino]- 2-Methyl-1-[[4-[6-(trifluoromethyl)-2-pyridinyl]-6-[[2-(trifluoromethyl)-4-pyridinyl]amino]-1,3,5-triazin-2-yl]amino]-2-propanol 2-Methyl-1-(4-(6-(trifluoromethyl)pyridin-2-yl)-6-(2-(trifluoromethyl)pyridin-4-ylamino)-1,3,5-triazin-2-ylamino)propan-2-ol   AG-221 CC-90007 1446502-11-9[RN] enasidenib Enasidenib énasidénib enasidenibum UNII:3T1SS4E7AG Энасидениб[Russian] إيناسيدينيب[Arabic] 伊那尼布[Chinese] 2-methyl-1-[(4-[6-(trifluoromethyl)pyridin-2-yl]-6-{[2-(trifluoromethyl)pyridin-4-yl]amino}-1,3,5-triazin-2-yl)amino]propan-2-ol 2-methyl-1-[[4-[6-(trifluoromethyl)pyridin-2-yl]-6-[[2-(trifluoromethyl)pyridin-4-yl]amino]-1,3,5-triazin-2-yl]amino]propan-2-ol 2-methyl-1-(4-(6-(trifluoromethyl)pyridin-2-yl)-6-(2-(trifluoromethyl)pyridin-4-ylamino)-1,3,5-triazin-2-ylamino)propan-2-ol Originator Agios Pharmaceuticals Developer Celgene Corporation Mechanism Of Action Isocitrate dehydrogenase 2 inhibitor Who Atc Codes L01 (Antineoplastic Agents) Ephmra Codes L1 (Antineoplastics) Indication Cancer …