Positive Phase 3 Results for Kalydeco Label-Expansion in Cystic Fibrosis

Vertex Pharmaceuticals announced the first clinical data from its ongoing Phase 3 label-expansion study of Kalydeco (ivacaftor) monotherapy in people with cystic fibrosis (CF) who have a non-G551D gating mutation. Kalydeco potentiates the cystic fibrosis transmembrane conductance regulator (CFTR) protein, a chloride channel, to regulate the transport of chloride ions and water in and out of cells.

The Phase 3 cross-over study of ivacaftor 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. Following the washout period, 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 absolute change from baseline in percent predicted FEV1 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. The mean absolute and relative percent predicted FEV1 improvements during ivacaftor treatment (within-group) were 7.5% (P<0.0001) and 10.8% (P<0.0001), respectively.

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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