FDA approves Kalydeco as first and only CFTR modulator to treat eligible infants with CF as early as four months of age.- Vertex

Vertex Pharmaceuticals Incorporated announced the FDA approved Kalydeco (ivacaftor) for use in children with cystic fibrosis (CF) ages four months to less than six months old who have at least one mutation in their cystic fibrosis transmembrane conductance regulator (CFTR) gene that is responsive to Kalydeco based on clinical and/or in vitro assay data. Kalydeco is already approved in the U.S. and EU for the treatment of CF in patients ages six months and older. This FDA approval is based on data from a cohort in the 24-week Phase III open-label safety cohort (ARRIVAL) consisting of 6 children with CF ages four months to less than six months who have one of 10 mutations in the CFTR gene (G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, G1349D or R117H). This cohort demonstrated a safety profile similar to that observed in older children and adults.