CHMP positive for Kalydeco to treat patients With cystic fibrosis aged 12 to less than 24 months With certain mutations in the CFTR gene.- Vertex.

Vertex Pharmaceuticals (Europe) Limited announces that the European Medicines Agency�s (EMA) Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion for Kalydeco (ivacaftor) to include the treatment of people with cystic fibrosis (CF) aged 12 to less than 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. If the European Commission issues a favorable adoption of the CHMP opinion for the extension of indication, ivacaftor will be the first and only medicine approved in Europe to treat the underlying cause of CF in patients aged 12 to less then 24 months, who have specific mutations in the CFTR gene.

The submission was supported by data from the ongoing Phase III open-label safety study (ARRIVAL) of children with CF aged 12 to less than 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.