EU approves extension of approval of Kalydeco to infants with CFTR cystic fibrosis.- Vertex Pharma

Vertex Pharmaceuticals announced that the European Commission has granted approval of the label extension for Kalydeco (ivacaftor) to include the treatment of infants with cystic fibrosis (CF) ages 6 months to less than 12 months weighing 5 kg and more 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 who are less than 24 months of age and have a CFTR gating mutation. The study showed a safety profile similar to that observed in previous Phase III studies of older children and adults, and improvements in sweat chloride, a secondary endpoint.

Comment: Kalydeco (ivacaftor) is already approved in Europe for the treatment of CF in patients ages 12 months and older who have one of the 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 18 years and older who have the R117H mutation in the CFTR gene.