On 3 May 2023, Vertex Pharmaceuticals have announced the U.S.Food and Drug Administration (FDA) approved KALYDECO (ivacaftor) for use in children with cystic fibrosis (CF) ages 1 month to less than four 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 four months and older.
The approval was supported by a cohort in the Phase 3, 24-week, open-label study to evaluate the safety, pharmacokinetics and pharmacodynamics of ivacaftor in subjects with CF who are less than 24 months of age and have an ivacaftor-responsive CFTR mutation.
This cohort demonstrated a safety profile similar to that observed in older children and adults.
