EC approval for ivacaftor extended to treat 12 to 24-month-old patients
Ivacaftor is the first and only approved medicine in Europe to treat the underlying cause of cystic fibrosis in these young patients
Vertex 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.
“For the first time, EU physicians can now treat the underlying cause of CF earlier than ever, helping to improve clinical outcomes in children as young as 12 months,” said Reshma Kewalramani, MD, Executive Vice President, Global Medicines Development and Medical Affairs and Chief Medical Officer at Vertex. “We look forward to supporting additional research on the potential benefit of early intervention with our medicines, with the goal of bringing treatment to all people living with CF.”
The label update is based on data from the ongoing Phase 3 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 3 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.