On Apr. 26, 2023, Vertex announced the Food and Drug Administration (FDA) had approved the expanded use of TRIKAFTA (elexacaftor/tezacaftor/ivacaftor and ivacaftor) to include children with cystic fibrosis (CF) ages 2 through 5 years who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene or a mutation in the CFTR gene that is responsive to TRIKAFTAﾮ based on in vitro data.

This label expansion was supported by a 24-week Phase 3 open-label study which enrolled 75 children ages 2 through 5 years old with CF to evaluate the safety, pharmacokinetics and efficacy of TRIKAFTA^®. The regimen was generally well tolerated, with a safety profile consistent with that observed in older age groups, and led to improvements in sweat chloride concentration, a measure of CFTR function, and lung function. The data from this study were recently published in the American Journal of Respiratory and Critical Care Medicine.

TRIKAFTA was previously approved by the FDA for use in people with CF 6 years and older with at least one F508del mutation or a mutation in the CFTR gene that is responsive to TRIKAFTAﾮ based on in vitro data.

TRIKAFTA^® was previously approved for the treatment of people with CF ages 6 years and older with certain mutations in the U.S., Canada, Switzerland, Australia, New Zealand and Israel, as well as in the EU, the U.K., Iceland, Liechtenstein and Norway as KAFTRIO^® (ivacaftor/tezacaftor/elexacaftor) in a combination regimen with KALYDECO^® (ivacaftor).

Additionally, Vertex has submitted applications for the use of TRIKAFTA^®/KAFTRIO^® in children ages 2 through 5 years of age to other global regulatory authorities, including the European Medicines Agency (EMA) and the Medicines and Healthcare Products Regulatory Agency (MHRA).