Vertex’s Kalydeco gets FDA aapproval for children with cystic fibrosis

The drug is approved for children with CF who have one of ten mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene (G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, S549R and R117H).

The approval is based on data from an open-label, 24-week Phase III trial that was designed to evaluate the safety and pharmacokinetics of weight-based dosing of ivacaftor (50mg or 75mg twice daily) in children of this age group.

Before securing this approval, the drug was approved in the US for people aged six years and older with these mutations.

Vertex executive vice-president and chief medical officer Jeffrey Chodakewitz said: "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.

"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."

Currently, more than 3,400 people are eligible for treatment with Kalydeco in the US, Canada, Europe and Australia.

In the US, there are about 300 children aged two to five years who have one of these ten 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.

In order to meet the needs of children in this age group who may be unable to swallow a tablet, a new weight-based oral granule formulation of Kalydeco (50mg and 75mg) was created that can be mixed in soft foods or liquids.