Vertex obtains FDA blessing for new Kalydeco indication

The Food and Drug Administration has given the green light to Vertex Pharmaceuticals for a new indication of its Kalydeco. The drug, first approved in 2012, now is indicated to treat children between 4 and 6 months old who have at least one mutation in their cystic fibrosis transmembrane conductance regulator gene that is responsive to Kalydeco based on clinical and/or in vitro assay data.

“Since the initial approval of Kalydeco more than eight years ago, we have continued to advance our clinical development program with the goal of treating the underlying cause of cystic fibrosis as early in life as possible,” said Reshma Kewalramani, Vertex CEO and president. “Today’s approval is a testament to our relentless efforts, alongside the clinical and scientific community, to reach all people with CF who may benefit from our medicines.”

“Initiating therapy that treats the underlying cause of cystic fibrosis as early as four months of age may have the potential to modify the course of the disease,” said Margaret Rosenfeld, Seattle Children’s Research Institute and Department of Pediatrics, University of Washington School of Medicine.

Kalydeco was initially approved for the treatment of CF in patients ages six months and older.