FDA adds more CF mutations to Kalydeco label

FDA expanded the label of cystic fibrosis drug Kalydeco ivacaftor from Vertex Pharmaceuticals Inc. (NASDAQ:VRTX) to include five additional disease-causing mutations in the CF transmembrane conductance regulator (CFTR) gene. The expanded label provides an option for over 600 CF patients, or about 2% the U.S. CF population.

Vertex said the five mutations are associated with “moderate loss of chloride transport." Kalydeco, a small molecule potentiator of CFTR, is now approved in patients ages two and older with any of 38 mutations in the CFTR gene...