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European Commission approves cystic fibrosis treatment for six to 11-year-olds

There is currently no cure for cystic fibrosis (CF), a debilitating, progressive condition with over 10,830 people in the UK currently diagnosed with the disease.

The European Commission (EC) has granted approval for the label extension of Kaftrio (ivacaftor/tezacaftor/elexacaftor), in a combination regimen with ivacaftor.

This has been recommended for the treatment of CF in patients aged six to 11-years-old who have at least one F508del mutation in the CF transmembrane conductance regulator (CFTR) gene.

The EC approval means that more than 1,500 children are now eligible to receive treatment for the underlying cause of CF for the first time.

Kaftrio, in combination with ivacaftor, is an oral medicine designed to increase the quality and function of the CFTR protein at the cell surface. These work together to increase the amount of mature protein at the cell surface by binding different sites on the CFTR protein.

Ivacaftor is designed to facilitate the ability of CFTR proteins to transport salt and water across the cell membrane. In combination, the medicines work together to help hydrate and clear mucus from the airways.

CF is a debilitating, life-shortening, inherited condition that causes progressive damage to organs across the body from birth. There is currently no cure for it and half of the people in the UK with CF die before the age of 36. Over 10,830 people in the UK are diagnosed with CF and this is the second-highest number in the world.

Treatment has a sign on the daily lives of patients, and this can take up to four or more hours involving nebulisers, physiotherapy, and up to 70 tablets a day.

“We are delighted that Kaftrio (ivacaftor/tezacaftor/elexacaftor) in a combination regimen with ivacaftor is now approved for these young patients in the EU. It provides a new treatment option for physicians to help treat the underlying cause of this devastating disease early in life,” said Reshma Kewalramani, CEO and president at Vertex. “This important milestone brings us one step closer to our ultimate goal of developing treatments for all patients living with CF.”