Massachusetts-based Vertex Pharmaceuticals announced positive results from a 24-week analysis of the ongoing phase III ENVISION study of VX-770, an oral medicine in development that targets the defective protein that causes cystic fibrosis (CF). ENVISION (n=52) was designed to evaluate VX-770 among children ages 6 to 11 with the G551D mutation in the CFTR gene.
The study met its primary endpoint of mean absolute change from baseline in percent predicted FEV1 (forced expiratory volume in one second, or lung function) through week 24. A difference in mean absolute improvement from baseline in lung function of 12.5% and a difference in mean relative improvement from baseline in lung function of 17.4 % compared to placebo (p
Vertex is on track to submit regulatory applications for approval in the U.S. and Europe in the second half of 2011. The submissions will be based on data from the phase III STRIVE and ENVISION studies as well as the phase II DISCOVER study. Vertex plans to submit data from all studies in the VX-770 phase III registration program for presentation at upcoming medical meetings.