Vertex today announced the submission of a supplemental New Drug Application (sNDA) to the FDA for the approval of Kalydeco (ivacaftor) 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. Kalydeco potentiates the CFTR protein, a chloride channel, to regulate the transport of chloride ions and water in and out of cells.
The sNDA submission is based on a Phase 3 cross-over study of ivacaftor monotherapy that enrolled 39 cystic fibrosis patients ages six and older with at least one non-G511D mutation. Patients received either ivacaftor, or placebo, for eight weeks, followed by a 4-week washout period, after which, patients who received placebo in the first eight weeks received ivacaftor for weeks 12–20, and patients who received ivacaftor for the first eight weeks received placebo for weeks 12–20. The primary endpoint of the study was absolute change from baseline in percent predicted FEV1.
The primary endpoint of this study was met. The mean absolute treatment difference in percent predicted FEV1 between treatment with ivacaftor and placebo was 10.7% (P<0.0001) and the mean relative treatment difference in percent predicted FEV1 was 14.2% (P<0.0001) through the 8-week treatment period.
As part of the sNDA package, Vertex also submitted long-term safety and efficacy data from the 96-week PERSIST open-label study of Kalydeco, 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 included efficacy and safety results through 144 weeks of continuous treatment with Kalydeco.
Kalydeco is already approved for the treatment of cystic fibrosis in people ages 6 and older who have at least one copy of the G551D mutation in the CFTR gene.
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