USA: The US Food and Drug Administration (FDA) has given approval to ivacaftor for treatment of cystic fibrosis in infants aged four months to less than six months who have at least one mutation in their cystic fibrosis transmembrane conductance regulator (CFTR) gene. Kalydeco (ivacaftor) is already approved by the FDA for treating cystic fibrosis in patients aged 6 months and older.

"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, M.D., MPH, Seattle Children's Research Institute and Department of Pediatrics, University of Washington School of Medicine.

This FDA approval is based on the results from 24-week Phase 3 open-label safety cohort (ARRIVAL) trial consisting of 6 children with CF ages four months to less than six months who have one of 10 mutations in the CFTR gene (G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, G1349D or R117H). This cohort demonstrated a safety profile similar to that observed in older children and adults.

"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, M.D., Chief Executive Officer and President, Vertex. "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."

About Cystic Fibrosis

Cystic fibrosis, a rare, progressive, life-threatening disease, results in the formation of thick mucus that builds up in the lungs, digestive tract, and other parts of the body. It leads to severe respiratory and digestive problems as well as other complications such as infections and diabetes. Cystic fibrosis is caused by a defective protein that results from mutations in the CFTR gene. While there are approximately 2,000 known mutations of the CFTR gene, the most common mutation is the F508del mutation.