Vertex Submits Supplemental New Drug Application (sNDA) To U.S. Food And Drug Administration For KALYDECO™ (ivacaftor) Monotherapy For People With Non-G551D Gating Mutations

Vertex Pharmaceuticals Incorporated (Nasdaq:VRTX) today announced the submission of a supplemental New Drug Application (sNDA) to the U.S. Food and Drug Administration (FDA) for the approval of KALYDECO TM (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 is currently approved for people with CF ages 6 and older who have at least one copy of the G551D mutation. CF is caused by a defective CFTR protein that results from mutations in the CFTR gene. G551D is known as a gating mutation, and there are an additional nine known gating mutations. Gating mutations prevent the CFTR protein from opening or working (gating) properly at the cell surface. Worldwide, approximately 2,000 people with CF ages 6 and older have at least one copy of the G551D mutation, and approximately 400 people with CF ages 6 and older have at least one non-G551D gating mutation.

"Today’s submission to the FDA is an important step toward our goal of helping more people with CF," said Robert Kauffman, M.D., Ph.D., Senior Vice President and Chief Medical Officer at Vertex. “The study in people with non-G551D gating mutations is the first of multiple ongoing efforts to expand the number of people who may benefit from ivacaftor, and we expect additional data from these ongoing studies beginning later this year.”

The sNDA submission is based on previously announced data from a Phase 3 study of ivacaftor monotherapy that showed statistically significant improvements in lung function (FEV 1). The mean absolute treatment difference in percent predicted FEV 1 between treatment with ivacaftor and placebo was 10.7% (p < 0.0001) and the mean relative treatment difference in percent predicted FEV 1 was 14.2% (p < 0.0001) through the 8-week treatment period. The safety and tolerability results observed in this study were consistent with those observed in prior Phase 3 studies of ivacaftor monotherapy in people with CF who have the G551D mutation. The study in gating mutations is one of three ongoing Phase 3 label-expansion studies designed to evaluate whether additional people with CF may benefit from treatment with ivacaftor alone.

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.

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