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 …
Tag «Orphan Drug Status»
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 …
BLU 285
It’s only fair to share… BLU-285 CAS 1703793-34-3 Molecular FormulaC26H27FN10 Average mass498.558 Da (1S)-1-(4-Fluorophenyl)-1-(2-{4-[6-(1-methyl-1H-pyrazol-4-yl)pyrrolo[2,1-f][1,2,4]triazin-4-yl]-1-piperazinyl}-5-pyrimidinyl)ethanamine 5-Pyrimidinemethanamine, α-(4-fluorophenyl)-α-methyl-2-[4-[6-(1-methyl-1H-pyrazol-4-yl)pyrrolo[2,1-f][1,2,4]triazin-4-yl]-1-piperazinyl]-, (αS)- 5-Pyrimidinemethanamine, α-(4-fluorophenyl)-α-methyl-2-[4-[6-(1-methyl-1H-pyrazol-4-yl)pyrrolo[2,1-f][1,2,4]triazin-4-yl]-1-piperazinyl]-, (αS)- Originator Blueprint Medicines Class Antineoplastics; Skin disorder therapies; Small molecules Mechanism of Action Platelet-derived growth factor alpha receptor modulators; Proto oncogene protein c-kit inhibitors Orphan Drug Status Yes – Systemic mastocytosis; Gastrointestinal stromal tumours Phase I Gastrointestinal stromal tumours; Solid tumours; …
VT 1129
It’s only fair to share… VT 1129 1340593-70-5 CAS MF C22 H14 F7 N5 O2, MW 513.37 2-Pyridineethanol, α-(2,4-difluorophenyl)-β,β-difluoro-α-(1H-tetrazol-1-ylmethyl)-5-[4-(trifluoromethoxy)phenyl]-, (αR)- R ISOMER ROTATION + Originator Viamet Pharmaceuticals Class Antifungals; Small molecules Mechanism of Action 14-alpha demethylase inhibitors Orphan Drug Status Yes – Cryptococcosis On Fast track Cryptococcosis Phase I Cryptococcosis Most Recent Events 01 Jun 2016 VT 1129 receives Fast Track designation for Cryptococcosis …
DROXIDOPA
It’s only fair to share… DROXIDOPA ORPHAN DRUG, CAS 23651-95-8, 3916-18-5 ROTATION –FORM (2S,3R)-2-amino-3-(3,4-dihydroxyphenyl)-3-hydroxypropanoic acid 223-480-5 [EINECS], L-Tyrosine, β,3-dihydroxy-, (βR)-, L-Tyrosine, β,3-dihydroxy-, threo- Northera [Trade name] threo-b,3-Dihydroxy-L-tyrosine 1-14-00-00685 [Beilstein], L-threo-3-(3,4-Dihydroxyphenyl)serine, L-Dihydroxyphenylserine, L-DOPS;DOPS, \ Northera, SM5688, L-DOPS|Northera®, |SM-5688, L-threo-dihydroxyphenylserine, L-Threodops L-threo-droxidopa, threo-2-Amino-3-(3,4-dihydroxyphenyl)-3-hydroxypropionic acid THREO-DIHYDROXYPHENYLSERINE threo-Dopaserine UNII:24A0V01WKS UNII:J7A92W69L7 FDA 2/18/2014, FDA approves Northera to treat neurogenic orthostatic hypotension Properties: Crystals from ethanol and ether, mp 232-235° (dec). [a]D20 -39° …
Evofosfamide, эвофосфамид , إيفوفوسفاميد , 艾伏磷酰胺 ,
It’s only fair to share… Evofosfamide, HAP-302 , TH-302, TH 302 эвофосфамид , إيفوفوسفاميد , 艾伏磷酰胺 , Molecular Formula C9H16Br2N5O4P Average mass 449.036 Da (1-Methyl-2-nitro-1H-imidazol-5-yl)methyl N,N’-bis(2-bromoethyl)phosphorodiamidate (1-Methyl-2-nitro-1H-imidazol-5-yl)methyl-N,N’-bis(2-bromethyl)phosphorodiamidat 918633-87-1 TH-302 is a nitroimidazole-linked prodrug of a brominated derivative of an isophosphoramide mustard previously used in cancer drugs Originator Threshold Pharmaceuticals Developer Merck KGaA; Threshold Pharmaceuticals Class Antineoplastics; Nitroimidazoles; Phosphoramide mustards; Small …