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

Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today 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.

About Cystic Fibrosis
Cystic fibrosis (CF) is a rare, life-shortening genetic disease affecting more than 88,000 people globally. CF is a progressive, multi-organ disease that affects the lungs, liver, pancreas, GI tract, sinuses, sweat glands and reproductive tract. CF is caused by a defective and/or missing CFTR protein resulting from certain mutations in the CFTR gene. Children must inherit two defective CFTR genes — one from each parent — to have CF, and these mutations can be identified by a genetic test. While there are many different types of CFTR mutations that can cause the disease, the vast majority of people with CF have at least one F508del mutation. CFTR mutations lead to CF by causing CFTR protein to be defective or by leading to a shortage or absence of CFTR protein at the cell surface. The defective function and/or absence of CFTR protein results in poor flow of salt and water into and out of the cells in a number of organs. In the lungs, this leads to the buildup of abnormally thick, sticky mucus, chronic lung infections and progressive lung damage that eventually leads to death for many patients. The median age of death is in the 30s, but with treatment, projected survival is improving.

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