On Nov. 23, 2023, Vertex announced that the European Medicines Agency (EMA) had validated a Type II variation application to the Marketing Authorization for KAFTRIOﾮ (ivacaftor/tezacaftor/elexacaftor) in combination with ivacaftor.

The application was for expansion of the approved indication for KAFTRIOﾮ in a combination regimen with ivacaftor for the treatment of people with cystic fibrosis (CF) ages 2 and above who have a mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene that is responsive based on clinical and/or in vitro data, including the N1303K mutation.