Vertex Pharmaceuticals announced that the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has issued a positive opinion for expanding the label of KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) in combination with ivacaftor to include people with cystic fibrosis (CF) aged 2 years and older with at least one non-class I CFTR mutation. If approved, around 4,000 additional CF patients in the European Union will have access to treatment targeting the disease’s underlying cause for the first time.

KAFTRIO is currently approved in the EU for patients with at least one copy of the F508del mutation in the CFTR gene. The therapy improves CFTR protein function, helping clear thick mucus from the lungs and improving lung function. Vertex’s CF medicines currently treat over 68,000 patients across more than 60 countries.

Cystic fibrosis is a progressive, life-shortening genetic disease affecting multiple organs. The median age of death is in the 30s, but treatment advances continue to improve projected survival. The CHMP's recommendation moves Vertex closer to its goal of expanding CF treatment access to all eligible patients.

The European Commission is expected to make a final decision on the label expansion in the coming months.