Vertex Pharmaceuticals has obtained approval from the European Commission (EC) for the label expansion of Kaftrio (ivacaftor/tezacaftor/elexacaftor) plus ivacaftor to treat children with cystic fibrosis (CF) aged between two and five years.

This combination therapy is intended for children who have at least one F508del mutation in the CF transmembrane conductance regulator (CFTR) gene.

CF is a progressive disease and impacts multiple organs, including the liver, lungs, gastrointestinal tract, pancreas, sinuses, sweat glands and reproductive tract. This life-shortening genetic disease affects more than 88,000 people across the world.

Kaftrio in combination with ivacaftor is an oral medicine that enhances the quantity and function of the CFTR protein at the cell surface.

Together, elexacaftor and tezacaftor increase the amount of mature protein at the cell surface by attaching to different sites on the CFTR protein.

Ivacaftor enhances the CFTR proteins’ ability to transport salt and water across the cell membrane.

Due to established re-imbursement agreements in Denmark, Austria, Ireland, Norway, Latvia and Sweden, patients who meet the criteria in these nations will benefit from the expanded approval for Kaftrio.

By collaborating with re-imbursement authorities throughout the European Union, Vertex will guarantee access for all qualifying patients.

Following the approval from the MHRA on 15 November 2023, and as a result of the re-imbursement agreement between the company and the NHS, children aged two years and older in the UK can now benefit from the extended use of Kaftrio.

Vertex global medicines development and medical affairs executive vice-president and chief medical officer Carmen Bozic 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.”