Vertex Pharma Says Phase 3 Study Results of Ivacaftor Did Not Meet Primary Endpoint

Vertex Pharmaceuticals Incorporated

today announced data from a Phase 3 study of ivacaftor in 69 people 6 years of age and older with cystic fibrosis (CF) who have the R117H mutation. In the study, the mean absolute treatment difference in the change from baseline in percent predicted FEV[1] between treatment with ivacaftor and placebo was 2.1 percentage points (p=0.20) and the mean relative treatment difference in percent predicted FEV[1] was 5.0 percent (p = 0.06) through the 24-week treatment period among all patients (intent-to-treat analysis). The study did not meet its primary endpoint of the absolute change from baseline in FEV[1] (percent predicted forced expiratory volume in one second, FEV[1]) throughout the treatment period for ivacaftor compared to placebo across all patients. A pre-specified subset analysis in patients 18 years of age and older (n=50) showed statistically significant improvements in lung function and other key secondary endpoints. In these patients, the mean absolute treatment difference in percent predicted FEV[1] between treatment with ivacaftor and placebo was 5.0 percentage points (p=0.01) and the mean relative treatment difference in percent predicted FEV[1] was 9.1 percent (p=0.008) through the 24-week treatment period. Vertex believes that the results show a clinical benefit for patients age 18 and older with the R117H mutation. The company plans to meet with the U.S. Food and Drug Administration (FDA) in early 2014 to discuss these data and the potential submission of a supplemental New Drug Application (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 United States, approximately 300 people have the R117H mutation and are 18 years of age or older.