Lumacaftor (VX-809) Chemical Properties

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)

pka

3.95±0.10(Predicted)

form 

Solid

color 

White to Off-White

Lumacaftor (VX-809) Usage And Synthesis

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

Lumacaftor (VX-809)(936727-05-8)Related Product Information

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