Vertex receives European CHMP positive opinion for KALYDECO
Vertex Pharmaceuticals announces that the European Medicines Agency's Committee for Medicinal Products for Human Use adopted a positive opinion for KALYDECO to include the treatment of people with cystic fibrosis aged 12 to less than24 months who have at least one of the following nine mutations in their cystic fibrosis transmembrane conductance regulator gene: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N or S549R. If the European Commission issues a favorable adoption of the EMA 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 than24 months, who have specific mutations in the CFTR gene. The submission was supported by data from the ongoing Phase 3 open-label safety study of children with CF aged 12 to less than24 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.