Vertex announced final results from the Phase 3 ENVISION study of Kalydeco (VX-770, ivacaftor), a drug being evaluated to target the G551D protein mutation that causes cystic fibrosis (CF). ENVISION (n=52) was designed to evaluate children with CF ages 6–11 years who had at least one copy of the G551D mutation. Results from ENVISION showed that children who received KALYDECO experienced a mean absolute improvement from baseline in lung function of 12.5 percentage points through Week 24 (primary study endpoint) and a mean relative improvement from baseline in lung function of 17.4% compared with placebo. Through 48 weeks, the mean absolute improvement in lung function for children treated with Kalydeco was 10 percentage points compared to placebo and the relative mean improvement was 15.1% from baseline compared to placebo.
Kalydeco is an oral tablet that directly targets the defective CFTR protein known to cause CF. Kalydeco increases the function of defective CFTR protein by increasing the gating activity, or ability to transport chloride ions across the cell membrane, of CFTR once it reaches the cell surface.
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