Vertex submits Kalydeco sNDA

Vertex Pharmaceuticals Inc. (NASDAQ:VRTX) submitted an sNDA to FDA for Kalydeco ivacaftor as monotherapy to treat cystic fibrosis (CF) in patients six years of age and older who have at least one non-G551D gating mutation in the cystic fibrosis transmembrane conductance regulatory ( CFTR) gene. The biotech plans to submit an MAA to EMA for the indication in October.

The small molecule potentiator of CFTR is approved in the U.S., Europe and Australia to treat CF in patients six years of age and older who have at least one copy of the G551D mutation in the CFTR gene -- about 4% of the CF population. Vertex said about 1% of the CF patients in the U.S. have at least one non-G551D gating mutation. ...