Vertex files for US priority review and approval of Kalydeco for cystic fibrosis

USA-based Vertex Pharmaceuticals (Nasdaq: VRTX) says it has filed a New Drug Application with the Food and Drug Administration 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 aged six and older who have at least one copy of the G551D mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. In the USA, approximately 4% 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. The majority of adverse events associated with the drug were mild to moderate in severity and non-serious. Fewer people in the Kalydeco treatment groups than in the placebo arms discontinued treatment due to adverse events. If approved, Kalydeco will be the first treatment to target the underlying cause of CF, said Vertex.