Kalydeco (ivacaftor) has been approved in the European Union for children ages 1-2 with cystic fibrosis (CF) who carry at least one of nine mutations in the CFTR gene, according to Vertex Pharma.
The approval was based on results from the ongoing Phase 3 ARRIVAL (NCT02725567) trial that enrolled children ages 1-2 with one of 10 CFTR mutations — G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, G1349D, or R117H.
The trial, conducted in two parts, first treated seven children with 50 mg or 75 mg of Kalydeco, depending on weight, every 12 hours for five days to assess safety. In the second part, nine children received 50 mg of Kalydeco for 24 weeks to evaluate its safety and efficacy.
Results showed that after 24 weeks, the children’s mean sweat chloride levels were about three times lower than those at the trial’s start — dropping from 104.1 mmol/L to a mean value of 33.8 mmol/L.