VX-661 is currently under investigation for the treatment of cystic fibrosis. VX-661 is a cystic fibrosis transmembrane conductance regulator modulator. Novel deuterated analogs of a cyclopropanecarboxamide ie tezacaftor (VX-661), as modulators of cystic fibrosis transmembrane conductance regulator (CFTR) proteins, useful for treating a CFTR-mediated disorder eg cystic fibrosis. VX-661 is an orally available deltaF508-CFTR corrector in phase III clinical trials at Vertex for the treatment of cystic fibrosis in patients homozygous to the F508del-CFTR mutation VX-661 is currently under Phase III clinical trial. VX-661 can correct trafficking of Δ508CFTR and partially restore chloride channel activity. The most common mutation, Δ508CFTR, produces a protein that is misfolded and does not reach the cell membrane. Cystic fibrosis (CF) is a genetic disease caused by defects in the CF transmembrane regulator (CFTR) gene, which encodes an epithelial chloride channel.
VX-661 is potentially useful for treatment of cystic fibrosis disease. Tezacaftor, also known asVX-661, is CFTR modulator.
In July 2016, this combination was reported to be in phase 3 clinical development. L-(2,2-difluoro-l,3-benzodioxol-5-yl)-N-6-fluoro-2-(2-hydroxy-l,l-dimethylethyl)-lH-indol-5-yl]-cyclopropanecarboxamide).
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