| Identification | Back Directory | [Name]
Lumacaftor (VX-809) | [CAS]
936727-05-8 | [Synonyms]
VX-809
CS-719
VRT 826809
Lumacaftor
3-(6-{[1-(2
VX809;VX-809
VX 809, >=98%
Lumacaftor
VX809
VX-809 + IVACAFTOR
VX-809 (LuMacaftor)
Lumacaftor(vx-809,vx809)
VX-809 (LUMACAFTOR);VX 809
VX-809, Lumacaftor, VRT 826809
3-(6-{[1-(2,2-Difluorobenzo[1,3]dioxol-5-yl)cyclopropanecarbonyl]-amino}-3-methyl-pyridin-2-yl
3-(6-{[1-(2,2-Difluorobenzo[1,3]dioxol-5-yl)cyclopropanecarbonyl]-amino}-3-methyl-pyridin-2-yl)be
3-(6-(1-(2,2-difluorobenzo[d][1,3]dioxol-5-yl)cyclopropanecarboxaMido)-3-Methylpyridin-2-yl)benzoic acid
3-[6-[[1-(2,2-difluoro-1,3-benzodioxol-5-yl)cyclopropanecarbonyl]amino]-3-methylpyridin-2-yl]benzoic acid
3-[6-[[[1-(2,2-Difluoro-1,3-benzodioxol-5-yl)cyclopropyl]carbonyl]amino]-3-methyl-2-pyridinyl]benzoic acid
3-(6-{[1-(2,2-Difluoro-benzo[1,3]dioxol-5-yl)-cyclopropanecarbonyl]-amino}-3-methyl-pyridin-2-yl)-benzoicacid
Benzoic acid, 3-[6-[[[1-(2,2-difluoro-1,3-benzodioxol-5-yl)cyclopropyl]carbonyl]amino]-3-methyl-2-pyridinyl]-
3-[6-[[[1-(2,2-Difluoro-1,3-benzodioxol-5-yl)cyclopropyl]carbonyl]amino]-3-methyl-2-pyridinyl]benzoic acid VX 809 Lumacaftor | [Molecular Formula]
C24H18F2N2O5 | [MDL Number]
MFCD16659051 | [MOL File]
936727-05-8.mol | [Molecular Weight]
452.407 |
| Chemical Properties | Back Directory | [Melting point ]
200-205oC | [Boiling point ]
653.0±55.0 °C(Predicted) | [density ]
1.51 | [storage temp. ]
-20°C Freezer | [solubility ]
DMSO (Slightly), Methanol (Slightly) | [form ]
Solid | [pka]
3.95±0.10(Predicted) | [color ]
White to Off-White | [InChIKey]
UFSKUSARDNFIRC-UHFFFAOYSA-N | [SMILES]
C(O)(=O)C1=CC=CC(C2=NC(NC(C3(C4=CC=C5OC(F)(F)OC5=C4)CC3)=O)=CC=C2C)=C1 |
| Hazard Information | Back Directory | [Uses]
VX 809 is used in the stabilization of the CFTR protein used in the treatment of cystic fibrosis. | [Definition]
ChEBI: An aromatic amide obtained by formal condensation of the carboxy group of 1-(2,2-difluoro-1,3-benzodioxol-5-yl)cyclopropane-1-carboxylic acid with the aromatic amino group of 3-(6-amino-3-methylpyridin-2-yl)benzoic acid. Used for the treatment of cystic fi
rosis. | [Biological Activity]
vx-809 is a cftr corrector that partially restores the function of f508del-cftr. in fischer rat thyroid (frt) cells, it increases f508del-cftr maturation at ec50 of 0.1 μm, and elevates f508del-cftr–mediated chloride transport at ec50 of 0.5 μm [1]. it has no effect of other ion channels (herg), transporter (p-gp) and disease-causing mislocalized proteins (α1-antitrypsin z mutant) [1]. vx-809 stabilizes n-terminal fragment of cftr that contain msd1 by altering its protein conformation [2, 3].homozygous f508del-cftr is the most common mutation in cystic fibrosis (cf) patients, accounting for 66–70% of cf cases worldwide. in cultured human bronchial epithelial cells that are homozygous for f508del, vx-809 restored the cftr function and improved chloride and fluid transport [1]. the combination of cftr potentiators and vx-809 further improved the function of f508del-cftr [4].vx-809 has been tested in several | [target]
CFTR | [storage]
Store at -20°C | [References]
[1]van goor f, hadida s, grootenhuis pd et al. correction of the f508del-cftr protein processing defect in vitro by the investigational drug vx-809. proc natl acad sci u s a 2011; 108: 18843-18848.
[2] ren hy, grove de, de la rosa o et al. vx-809 corrects folding defects in cystic fibrosis transmembrane conductance regulator protein through action on membrane-spanning domain 1. mol biol cell 2013; 24: 3016-3024.
[3]loo tw, bartlett mc, clarke dm. corrector vx-809 stabilizes the first transmembrane domain of cftr. biochem pharmacol 2013; 86: 612-619.
[4]clancy jp, rowe sm, accurso fj et al. results of a phase iia study of vx-809, an investigational cftr corrector compound, in subjects with cystic fibrosis homozygous for the f508del-cftr mutation. thorax 2012; 67: 12-18.
[5]http://investors.vrtx.com/releasedetail.cfm?releaseid=687394 |
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