KAFTRIO combination with ivacaftor receives regulatory approval for label expansion in Europe; ALYFTREK gets the green light in the UK; Zimislecel progressing through pivotal Phase 3 trial while VX-264 development is being discontinued.

On April 7, 2025, Vertex Pharmaceuticals announced that the European Commission granted final regulatory approval for the label expansion of cystic fibrosis medication KAFTRIO in combination with ivacaftor to include rare mutations.

The label for the combination treatment now includes patients with cystic fibrosis (CF) aged two years and older who have at least one non-class I mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, making approximately 4,000 additional CF patients across Europe newly eligible for a CFTR modulator. A non-class I CFTR gene mutation, in contrast to Class I mutations that lead to little or no CFTR protein production, encompasses mutations in ClassesII-VI that result in the CFTR protein being produced, but with some degree of dysfunction.

In the EU, KAFTRIO in combination with ivacaftor was previously approved only for the treatment of people with CF aged two years and older who have at least one copy of the F508del mutation in the CFTR gene.

The April 7 development followed a review by the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP), which had adopted a positive opinion on the label expansion on February 28, 2025.