European Commission Expands Approval for Vertex’s KAFTRIO® to Treat More Children With Cystic Fibrosis

Vertex Pharmaceuticals announced that the European Commission has approved an expanded label for KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) combined with ivacaftor. The approval now includes all patients aged two years and older with at least one non-class I CFTR gene mutation. This expansion means approximately 4,000 additional patients in the EU are now eligible for a treatment that targets the underlying cause of their cystic fibrosis (CF) for the first time.

KAFTRIO, already used by over 68,000 people across more than 60 countries, is designed to restore CFTR protein function and improve outcomes such as lung function and quality of life. Vertex confirmed that countries with existing reimbursement agreements, such as Germany, Sweden, and Austria, will soon offer access to the expanded indication. The company is also working to broaden access across the EU.

Cystic fibrosis is a rare genetic disease that affects multiple organs and is often fatal. The expanded approval represents a significant step forward in reaching underserved patients with rare CF mutations.