Europe's Committee for Medicinal Products for Human Use (CHMP) recommends that the European Medicines Agency (EMA) approve Vertex Pharmaceuticals' (VRTX) Kalydeco (ivacaftor) for cystic fibrosis (CF) patients ages six and older who have one of eight non-G551D gating mutations in the CF transmembrane conductance regulator ((CFTR)) gene. In July 2012, the EMA approved Kalydeco for CF patients with at least one copy of the G551D mutation.
CF is caused by a defective or missing CFTR protein that results from mutations in the CFTR gene. CFTR proteins act as channels at the cell surface that controls the flow of salt and water in and out of the cell. The dysfunction in CF causes mucus to build up in the lungs and affects the digestive tract and other organs. Kalydeco targets the abnormal CFTR channels and opens them up to allow chloride ions to move in and out of the cell. This thins the mucus so it can hydrate and protect the airways.