Vertex announces European Commission approval for ivacaftor (Kalydeco) to treat eligible infants with cystic fibrosis as early as four months of age
Approval granted for label extension for ivacaftor granules to include treatment of infants ages ≥4 months and weighing ≥5 kg with r117h mutation or one of a number of gating (class III) mutations in CFTR gene: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N or S549R.
Source:
Biospace Inc.
SPS commentary:
Approval is based on data from a cohort in the 24-week Phase 3 open-label safety study (ARRIVAL) consisting of 6 children.
Ivacaftor) is already approved in Europe for people ages 6 months and older weighing at least 5kg.