Posted by Michael Wonder on 11 Dec 2019
Vertex announces European Commission approval for Kalydeco (ivacaftor) in infants with cystic fibrosis ages 6 months to less than 12 months with certain mutations in the CFTR gene
10 December 2019 - Ivacaftor is the first and only approved medicine in Europe to treat the underlying cause of cystic fibrosis in children this young.
Vertex Pharmaceuticals today announced that the European Commission has granted approval of the label extension for Kalydeco (ivacaftor) to include the treatment of infants with cystic fibrosis 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 3 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 3 studies of older children and adults, and improvements in sweat chloride, a secondary endpoint.
