Vertex Files Kalydeco sNDA

Vertex Pharmaceuticals has submitted an sNDA to FDA for the approval of Kalydeco monotherapy for people with cystic fibrosis (CF) ages 6 and older who have at least one non-G551D gating mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. In addition to the sNDA submission, Vertex intends to submit a Marketing Authorization Application (MAA) variation in Europe in October 2013 for people with CF ages 6 and older who have at least one non-G551D gating mutation.

Kalydeco is currently approved for people with CF ages 6 and older who have at least one copy of the G551D mutation. The sNDA submission is based on previously announced data from a Phase III study of ivacaftor monotherapy that showed statistically significant improvements in lung function (FEV1). The mean absolute treatment difference in percent predicted FEV1 between treatment with ivacaftor and placebo was 10.7%.

As part of the sNDA package, Vertex also submitted long-term data from the 96-week PERSIST open-label study of Kalydeco. PERSIST is an ongoing rollover study of people with cystic fibrosis ages 6 and older with a G551D mutation who took part in the 48-week Phase 3 STRIVE and ENVISON studies of Kalydeco. The data from PERSIST submitted as part of the sNDA included efficacy and safety results through 144 weeks of continuous treatment with Kalydeco.