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GEN News Highlights : Mar 29, 2011

Phase III Study Shows Vertex Drug Significantly Boosts Lung Function in Children with CF

Data supports Strive study in adult patients with G551D mutation.

Pediatric cystic fibrosis patients treated using Vertex Pharmaceuticals’ oral CFTR potentiator VX-770 demonstrated significant improvements in lung function after 24 weeks of treatment, according to data from an ongoing Phase III study. Results from the completed 48-week Envision study will form part of the regulatory packages for VX-770 in the U.S. and Europe, which are on track for submission during the second half of 2011, the firm says.

The placebo-controlled Envision study is evaluating oral VX-770 in 52 children aged 6–11 years who carry at least one copy of the G551D CFTR gene mutation. Results at 24 weeks showed a mean absolute improvement from baseline in FEV1 of 12.5% and a difference in mean relative improvement from baseline in lung function of 17.4% compared with placebo. Treatment with VX-770 also led to significant improvements in other disease measures, including weight gain and reduction in sweat chloride.

“In this study, children with CF treated with VX-770 showed the same profound improvements in lung function seen in the recently announced Strive study among an older group of people with the G551D mutation,” comments Robert Kauffman, M.D., svp and CMO at Vertex.

The midway data from Envision support those from the Strive study in adult patients, which Vertex reported in February. The Cystic Fibrosis Foundation says results from the pediatric study are particularly encouraging because younger patients tend to be healthier than adults, and significant improvements in lung function in children can be harder to demonstrate.