EC Greenlights KAFTRIO® Combo for Cystic Fibrosis

Vertex Pharmaceuticals Incorporated has announced that the European Commission (EC) has approved 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.

Carmen Bozic, M.D., Executive Vice President, Global Medicines Development and Medical Affairs, and Chief Medical Officer, Vertex, stated:

“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.”

Professor Marcus A. Mall, M.D., Head of the Department of Pediatric Respiratory Medicine, Immunology and Critical Care Medicine at Charité Universitätsmedizin Berlin, stated:

“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.”

Eligible patients in Austria, Denmark, Ireland, Norway, Latvia, and Sweden will soon access the extended use of KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) in combination with ivacaftor, following European Commission approval. Vertex is actively collaborating with reimbursement authorities across the EU to ensure broad accessibility. In the U.K., MHRA approval on November 15, 2023, and an existing reimbursement agreement with the NHS enable children aged 2 and above can access the expanded indication for KAFTRIO®.