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Vertex Pharmaceuticals Inc Submits Supplemental New Drug Application To U.S. Food and Drug Administration for KALYDECO (ivacaftor) Monotherapy For People With Non-G551D Gating Mutations


Tuesday, 1 Oct 2013 09:32am EDT 

Vertex Pharmaceuticals Inc announced the submission of a supplemental New Drug Application (sNDA) to the U.S. Food and Drug Administration (FDA) for the approval of KALYDECOTM (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. 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 (FEV1). 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. 

Company Quote

0.45
-0.02 -4.26%
11 Nov 2013