Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced the first clinical data from one of the company’s ongoing Phase 3 label-expansion studies for ivacaftor monotherapy in people with cystic fibrosis (CF). In the Phase 3 study evaluating ivacaftor monotherapy in people ages six and older with at least one non-G551D gating mutation, 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. KALYDECO (ivacaftor) is currently approved for people with cystic fibrosis (CF) ages 6 and older who have at least one copy of the G551D mutation in the cystic fibrosis transmembrane conductance regulator ( CFTR) gene. Worldwide, approximately 2,000 people with CF ages six and older have at least one copy of the G551D mutation, and approximately 400 people with CF ages six and older have at least one non-G551D gating mutation. The study in gating mutations is one of three ongoing Phase 3 label-expansion studies for ivacaftor designed to evaluate whether additional people with CF may benefit from treatment with ivacaftor.
Based on these data, Vertex plans to submit a supplemental New Drug Application (sNDA) in the United States and a Marketing Authorization Application (MAA) variation in Europe in the second half of 2013 for the use of ivacaftor monotherapy in people with CF ages 6 and older who have at least one non-G551D CFTR gating mutation.
“Our goal in CF is to help as many people as possible with our medicines, and these data are an important step toward that goal,” said Robert Kauffman, M.D., Ph.D., Senior Vice President and Chief Medical Officer at Vertex. “The data announced today in people with CF who have a gating mutation showed a meaningful improvement in lung function and support our plans to seek approval of ivacaftor for these patients later this year.”