Vertex Pharmaceuticals Incorporated announced the submission of a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for KALYDECO™ (VX-770, ivacaftor), a medicine in development that targets the defective protein that causes cystic fibrosis (CF). KALYDECO (kuh-LYE-deh-koh) was studied among people with CF ages 6 and older who have at least one copy of the G551D mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. In the United States, approximately 4 percent of people with CF are estimated to have at least one copy of the G551D mutation in the CFTR gene.
Global Phase 3 pivotal studies of KALYDECO showed significant and sustained improvements in lung function and other measures of disease in people with CF who had at least one copy of the G551D mutation. The majority of adverse events associated with KALYDECO were mild to moderate in severity and non-serious. Fewer people in the KALYDECO treatment groups than in the placebo groups discontinued treatment due to adverse events. If approved, KALYDECO will be the first treatment to target the underlying cause of CF.
The U.S. submission includes a request for Priority Review, which, if granted, would shorten the FDA’s anticipated review time from 10 to six months. The FDA grants Priority Review status for several reasons, including if the medicine is considered a major advance in treatment. Vertex also plans to submit a marketing authorization application (MAA) for KALYDECO with the European Medicines Agency (EMA) by the end of October 2011. The EMA has accepted Vertex’s request for accelerated assessment, which is granted to new medicines of major public health interest and shortens the review time from 210 days to 150 days following the start of the review. Additionally, Vertex submitted requests to the FDA and EMA to use the trade name KALYDECO (ivacaftor) for VX-770.