European Commission Approves KAFTRIO in Combination With Ivacaftor for the Treatment of Children With Cystic Fibrosis Ages 2 Through 5

Vertex Pharmaceuticals Incorporated announced that the European Commission has granted approval for the label expansion of KAFTRIO (ivacaftor/tezacaftor/elexacaftor) in a combination regimen with ivacaftor for the treatment of children with cystic fibrosis (CF) ages 2 through 5 years old who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.

“In addition to data from clinical trials, long-term and real-world data have demonstrated the significant clinical benefit of KAFTRIO in eligible people living with CF, and today’s news means that young children across Europe can now benefit from this important medicine,” said Carmen Bozic, M.D., Executive Vice President, Global Medicines Development and Medical Affairs, and Chief Medical Officer, Vertex.

“As CF starts in early childhood and is a progressive disease, it is important to treat people with CF as early as possible. With the approval of KAFTRIO for children as young as 2 years, we can now treat young children with a medicine that has the potential to slow disease progression by addressing the underlying cause of the disease,” said Professor Marcus A. Mall, M.D., Head of the Department of Pediatric Respiratory Medicine, Immunology and Critical Care Medicine at Charité Universitätsmedizin Berlin.

As a result of existing reimbursement agreements in Austria, Denmark, Ireland, Norway, Latvia, and Sweden, eligible patients in these countries will have access to the expanded indication for KAFTRIO (ivacaftor/tezacaftor/elexacaftor) in a combination regimen with ivacaftor shortly following regulatory approval by the European Commission. Vertex will continue to work with reimbursement authorities across the European Union to ensure access for all eligible patients. In the U.K., following MHRA approval on November 15, 2023, and as a result of the existing reimbursement agreement between Vertex and the NHS, children ages 2 years and above in the U.K. have access to this expanded indication for KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) in a combination regimen with ivacaftor.