Phase 3 study of VX-770 for the treatment of cystic fibrosis
Vertex announced results from its Phase 3 study of VX-770 for the treatment of CF caused by a mutation in the G551D gene. This study, also known as the STRIVE trial, enrolled 161 patients with at least one copy of the G551D CF transmembrane conductance regulator (CFTR) mutation. The primary efficacy endpoint of the study was mean absolute change from baseline compared with placebo in percent predicted FEV1 (lung function) through Week 24. Data from the study showed a mean absolute improvement in lung function from baseline compared with placebo through Week 24 of 10.6% among those treated with VX-770.
VX-770 is an oral tablet that directly targets the defective CFTR protein known to cause CF. VX-770 increases the function of defective CFTR protein by increasing the gating activity, or ability to transport ions across the cell membrane, of CFTR once it reaches the cell surface.
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