Vertex’s Kalydeco Gets Additional FDA Approval for Cystic Fibrosis

Vertex Pharmaceuticals’ cystic fibrosis drug Kalydeco has won FDA approval for an additional indication to treat patients six years and older who have at least one copy of the R117H gene mutation.

Kalydeco (ivacaftor) initially was approved in 2012 as the first therapy to address the underlying defect in the CFTR gene that causes CF’s decreased or absent transport of salt out of cells. That approval was for patients who possessed at least one G551D mutation — only about 4 percent of the global CF population.

The latest approval adds to the roughly 10 other approved indications to treat CF gene mutations since the drug first came to market, Vertex says. Before Kalydeco, only maintenance treatments, such as antibiotics to treat constant lung infections or inhaled saline to thin lung mucus, were available for CF patients. — Lena Freund