Vertex Pharmaceuticals Incorporated Submits Application for Priority Review and Approval of KALYDECO(TM) (VX-770, ivacaftor) in the U.S. as First Potential Medicine to Target the Underlying Cause of Cystic Fibrosis

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today 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.