The US Food and Drug Administration (FDA) approved Vertex Pharmaceuticals' Kalydeco for use in children ages 2 to 5 with cystic fibrosis (CF) who have one of 10 mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene (G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, S549R and R117H).

Prior to today's approval, Kalydeco was approved in the United States for people ages 6 and older with these mutations. There are approximately 300 children in the United States ages 2 to 5 who have one of these 10 mutations, including 150 who have the R117H mutation and 150 who have one of the other nine mutations that result in a gating defect in the CFTR protein. A new weight-based oral granule formulation of Kalydeco (50 mg and 75 mg) that can be mixed in soft foods or liquids was created to meet the needs of children in this age group who may be unable to swallow a tablet.

The approval is based on previously announced results of an open-label phase 3 24-week study that was designed to evaluate the safety and pharmacokinetics of weight-based dosing of ivacaftor (50 mg or 75 mg twice daily) in children ages 2 to 5. With today's approval, more than 3,400 people are currently eligible for treatment with Kalydeco in the United States, Canada, Europe and Australia. Cystic fibrosis is caused by a defective or missing CFTR protein resulting from mutations in the CFTR gene.

"Children with cystic fibrosis can begin to experience meaningful lung function decline and struggle to gain weight at a very young age, underscoring the importance of starting treatment early in life," said Jeffrey Chodakewitz, M.D., executive vice president and chief medical officer at Vertex. "With today's approval, children as young as two years of age now have a medicine to treat the underlying cause of their CF, bringing us one step closer to our goal of helping the vast majority of people with this devastating disease."

In Europe, an MAA line extension for ivacaftor in children ages 2 to 5 with specific mutations in the CFTR gene has been validated by the European Medicines Agency (EMA) and is currently under review by the Committee for Medicinal Products for Human Use (CHMP).

Kalydeco (ivacaftor) is the first medicine to treat the underlying cause of CF in people with specific mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Known as a CFTR potentiator, Kalydeco is an oral medicine designed to keep CFTR proteins at the cell surface open longer to improve the transport of salt and water across the cell membrane, which helps hydrate and clear mucus from the airways.

Kalydeco is approved in the US, Europe, Canada, Australia and New Zealand to treat people with CF who have specific genetic mutations in the CFTR gene.

Vertex retains worldwide rights to develop and commercialize Kalydeco.

Cystic fibrosis is a rare, life-threatening genetic disease affecting approximately 75,000 people in North America, Europe and Australia. Today, the median predicted age of survival for a person with CF is between 34 and 47 years, but the median age of death remains in the mid-20s.

Vertex is a global biotechnology company that aims to discover, develop and commercialize innovative medicines so people with serious diseases can lead better lives.