European Commission approval for Kaftrio in combination with Ivacaftor to treat cystic fibrosis patients 12 years and older with at least one F508del mutation in the CFTR gene.- Vertex

Vertex Pharmaceuticals Incorporated announced that the European Commission has granted approval of the label extension for Kaftrio (ivacaftor/tezacaftor/elexacaftor) in a combination regimen with ivacaftor for the treatment of cystic fibrosis (CF) in all patients ages 12 years and older who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. With this extension of the indication, CF patients 12 years and older who are heterozygous for the F508del-CFTR mutation and a residual function (F/RF) or gating mutation (F/G) are eligible for the triple combination therapy for the first time, and the majority of people ages 12 years and older with CF in Europe are now eligible for Kaftrio. In both the United States and Australia, where the triple combination therapy is known as Trikafta (elexacaftor/tezacaftor/ivacaftor and ivacaftor), it is already licensed for the treatment of people with CF ages 12 years and older who have at least one copy of the F508del mutation in the CFTR gene, regardless of the other mutation type.