The US FDA has approved ivacaftor (Kalydeco, Vertex Pharmaceuticals) for children aged two to five years with cystic fibrosis (CF) who have one of 10 mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.

These mutations are: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, S549R, and R117H. Ivacaftor is already approved in the US for people aged 6 years or older with these mutations.

The expanded approval is based on results of an open-label, phase 3 study that evaluated the safety and pharmacokinetics of weight-based dosing of ivacaftor (50 mg or 75 mg twice daily) in two- to five-year-old children, Vertex noted in a news release.