Kalydeco ivacaftor: Phase III data

The double-blind, crossover, international Phase III KONNECTION trial in 39 CF patients >=6 years of age with at least 1 non-G551D CFTR gating mutation showed that twice-daily 150 mg oral Kalydeco met the primary endpoint of improving the mean absolute change in percent predicted FEV1 from baseline to week 8 vs. placebo. Specifically, the mean absolute improvement in percent predicted FEV1 at week 8 compared to placebo was 10.7% (p<0.0001). Kalydeco also led to a mean relative improvement in percent predicted FEV1 from baseline to week 8 of 14.2% compared to placebo (p<0.0001). On secondary endpoints, Kalydeco significantly improved weight gain and patient-reported quality of life as measured by the respiratory domain of the CFQ-R vs. placebo. Patients received Kalydeco or placebo for 8 weeks followed by a 4-week washout period. Following the washout period, patients crossed over to the other treatment arm for an additional 8 weeks. All patients received open-label Kalydeco from week 20 to 36. Final data will be submitted for presentation at a medical meeting this half. Also this half, Vertex plans to submit an sNDA to FDA and a variation to an MAA to EMA to expand the label for Kalydeco to include monotherapy use in CF patients >=6 years of age who have >=1 non-G551D CFTR gating mutation. According to Vertex, this population is about 400 patients worldwide. ...