Name: Tezacaftor (VX-661)
Chemical Formula: C26H27F3N2O6
Exact Mass: 520.18212
Molecular Weight: 520.5
Elemental Analysis: C, 60.00; H, 5.23; F, 10.95; N, 5.38; O, 18.44
Description: Tezacaftor, also known asVX-661, is CFTR modulator. VX-661 is potentially useful for treatment of cystic fibrosis disease. Cystic fibrosis (CF) is a genetic disease caused by defects in the CF transmembrane regulator (CFTR) gene, which encodes an epithelial chloride channel. The most common mutation, Δ508CFTR, produces a protein that is misfolded and does not reach the cell membrane. VX-661 can correct trafficking of Δ508CFTR and partially restore chloride channel activity.