EU extends paediatric use of combination in cystic fibrosis

Mother helps child to use a nebuliser

A label expansion for Kaftrio (ivacaftor/tezacaftor/elexacaftor) in a combination regimen with ivacaftor for the treatment of cystic fibrosis (CF) has been approved in the European Union (EU).

The European Commission has extended use of the therapy to include children with CF aged two through five years old who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.

“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 two 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 Mall, Head of the Department of Paediatric Respiratory Medicine, Immunology and Critical Care Medicine at Charité Universitätsmedizin Berlin.

Manufacturer Vertex has said it will continue to work with reimbursement authorities across the EU to ensure access for all eligible patients.

In the UK, following Medicines and Healthcare products Regulatory Agency (MHRA) approval on 15 November 2023, and as a result of the existing reimbursement agreement between Vertex and the NHS, children aged two years and above in the UK have access to this expanded indication for Kaftrio in a combination regimen with ivacaftor.

This follows the news that the MHRA was the first regulatory authority to authorise Vertex’s gene therapy for sickle-cell disease (SCD) and transfusion-dependent β-thalassemia (TDT).

Cystic fibrosis mutations

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 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.

“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, Executive Vice President, Global Medicines Development and Medical Affairs, and Chief Medical Officer, Vertex.

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