Kalydeco is filed at FDA and EMA for Cystic Fibrosis

The FDA has accepted the New Drug Application (NDA) for Kalydeco (ivacaftor) from Vertex Pharmaceuticals and granted the company's request for six-month Priority Review. Kalydeco targets the defective protein that causes Cystic Fibrosis (CF) in a subset of people with the disease. A target review date of April 18, 2012 is set. The EMA has validated the marketing authorization application (MAA) for Kalydeco and has given the application an accelerated assessment. CF is caused by defective or missing cystic fibrosis transmembrane conductance regulator (CFTR) proteins resulting from mutations in the CFTR gene. The absence of functional CFTR proteins results in poor flow of salt and water across cell membranes in a number of organs, including the lungs. This leads to the buildup of abnormally thick, sticky mucus that can cause chronic lung infections and progressive lung damage. In people with CF who have a gating defect, CFTR proteins are present at the cell surface but do not function properly. The most common gating defect is caused by the G551D mutation. Approximately 4 percent of those with CF, or about 1,200 people in the United States and 1,000 people in Europe , are believed to have this mutation. Kalydeco is designed to keep the CFTR channels at the cell surface open longer to improve the transport of chloride ions across the cell membrane in people who have gating mutations. The U.S. application seeks approval for Kalydeco in people with the G551D mutation. The application submitted in Europe includes a request for all gating mutations.