European Commission approves Vertex Pharma’s Kaftrio in combo with ivacaftor to treat 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 paediatric 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 UK, 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 UK have access to this expanded indication for Kaftrio (ivacaftor/tezacaftor/elexacaftor) in a combination regimen with ivacaftor.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.In people with certain types of mutations in the CFTR gene, the CFTR protein is not processed or folded normally within the cell, and this can prevent the CFTR protein from reaching the cell surface and functioning properly. Kaftrio (ivacaftor/tezacaftor/elexacaftor) in combination with ivacaftor is an oral medicine designed to increase the quantity and function of the CFTR protein at the cell surface. Elexacaftor and tezacaftor work together to increase the amount of mature protein at the cell surface by binding to different sites on the CFTR protein. Ivacaftor, which is known as a CFTR potentiator, is designed to facilitate the ability of CFTR proteins to transport salt and water across the cell membrane. The combined actions of ivacaftor, tezacaftor and elexacaftor help hydrate and clear mucus from the airways.Kaftrio (ivacaftor/tezacaftor/elexacaftor) in combination with ivacaftor is approved in the European Union for the treatment of cystic fibrosis (CF) in patients aged 2 years and older who have at least one copy of the F508del mutation in the CFTR gene.Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases.

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