Vertex announces results of Phase 3 Ivacaftor study Vertex Pharmaceuticals announced data from a Phase 3 study of ivacaftor in 69 people 6 years of age and older with cystic fibrosis who have the R117H mutation. 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 percentage points and the mean relative treatment difference in percent predicted FEV1 was 5.0% through the 24-week treatment period among all patients. The study did not meet its primary endpoint of the absolute change from baseline in FEV1 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 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 percentage points and the mean relative treatment difference in percent predicted FEV1 was 9.1 percent 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 in early 2014 to discuss these data and the potential submission of a supplemental New Drug Application for people with the R117H mutation.
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