Kalydeco filed at FDA for Cystic Fibrosis treatment

Vertex Pharmaceuticals has submitted a New Drug Application (NDA) to the FDA for Kalydeco (VX-770, ivacaftor), a medicine in development that targets the defective protein that causes cystic fibrosis (CF). Kalydeco 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 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 III 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. Kalydeco will be the first treatment to target the underlying cause of CF. Vertex Pharma intends to apply in October this year for EU approval. Applications are based on the positive results of STRIVE and ENVISION studies. 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.