Vertex CF Treatment Misses Endpoint

Vertex Pharmaceuticals has posted data from a Phase III study of ivacaftor in 69 people 6 years of age and older with cystic fibrosis (CF) who have the R117H mutation. The trial missed its primary endpoint of the absolute change from baseline in FEV1 (percent predicted forced expiratory volume in one second, FEV1) throughout the treatment period for ivacaftor compared to placebo across all patients.

In the study, the mean absolute treatment difference in the change from baseline in percent predicted FEV1 between treatment with ivacaftor and placebo was 2.1% and the mean relative treatment difference in percent predicted FEV1 was 5.0% through the 24-week treatment period among all patients (intent-to-treat analysis).

A pre-specified subset analysis in patients 18 years of age and older showed statistically significant improvements in lung function and other key secondary endpoints. In these patients, the mean absolute treatment difference in percent predicted FEV1 between treatment with ivacaftor and placebo was 5.0% and the mean relative treatment difference in percent predicted FEV1 was 9.1% through the 24-week treatment period.

The company plans to meet with the FDA in early 2014 to discuss these data and the potential submission of an sNDA for people with the R117H mutation. R117H is the most common residual function mutation. In North America, Europe and Australia, approximately 1,100 people with CF ages 6 and older have at least one copy of an R117H mutation. In the U.S. approximately 300 people have the R117H mutation and are 18 years of age or older.