EU Expands Approval of Vertex’s KAFTRIO to Treat More Young Cystic Fibrosis Patients

Vertex Pharmaceuticals announced that the European Commission has approved an expanded label for KAFTRIO (ivacaftor/tezacaftor/elexacaftor) in combination with ivacaftor. The treatment is now authorized for cystic fibrosis patients aged 2 and older with at least one non-class I CFTR gene mutation. This expansion means around 4,000 additional individuals in the EU are now eligible for a therapy that targets the underlying cause of the disease.

Existing reimbursement agreements in countries like Austria, Denmark, and Sweden will facilitate swift access, and Vertex plans to work with other EU health authorities to broaden availability. KAFTRIO helps improve CFTR protein function, enhancing the flow of salt and water in affected organs and ultimately easing symptoms such as thick mucus and lung infections. Vertex’s CF treatments now serve over 68,000 patients globally, two-thirds of those eligible for CFTR modulators.