European Commission approves label extension for Kalydeco to treat children with cystic fibrosis. Vertex

Vertex Pharmaceuticals (Europe) Limited announced that the European Commission has granted approval of the label extension for kalydeco (ivacaftor) to include the treatment of people with cystic fibrosis (CF) aged 12 to <24 months who have at least one of the following nine mutations in their cystic fibrosis transmembrane conductance regulator cftr gene: g551d g1244e g1349d g178r g551s s1251n s1255p s549n or s549r.>

The label update is based on data from the ongoing Phase III open-label safety study (ARRIVAL) of children with CF aged 12 to <24 months who have one of 10 mutations in the cftr gene that demonstrated a safety profile consistent with that observed in previous phase iii studies of older children and adults and improvements in sweat chloride a key secondary efficacy endpoint.>

Ivacaftor is already approved in Europe for the treatment of CF in patients aged two years and older who have one of the nine following mutations in the CFTR gene: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N or S549R. It is also approved for the treatment of CF in patients aged 18 years and older who have an R117H mutation in the CFTR gene..