FDA approves new indication for Kalydeco

SILVER SPRING, Md. — The Food and Drug Administration on Tuesday approved a new indication for Vertex Pharmaceuticals’ Kalydeco (ivacaftor). The new indication is for use in more than 600 people with cystic fibrosis ages 2 years and older who have one of five residual function mutations.

“In the five years since KALYDECO became the first approved medicine to treat the underlying cause of cystic fibrosis, we have been relentless in our efforts to bring this important medicine to all who may benefit,” Vertex chief medical officer Dr. Jeffrey Chodakewitz said. “We will continue to pursue this goal until all people with CF have a medicine that treats their form of this serious and life-shortening disease.”

The drug now is approved in the United States to treat cystic fibrosis patients ages 2 years and older with one of 28 ivacaftor-responsive mutations in the cystic fibrosis transmembrane conductance regulator, or CFTR, the company said.

The illness is caused by a defective or missing CFTR protein resulting from mutations in the CFTR gene. There are approximately 2,000 known mutations in the CFTR gene. Some of these mutations, which can be determined by a genetic test, lead to CF by creating defective or too few CFTR proteins at the cell surface.