Cystic fibrosis drug to be reimbursed for children aged 2 to 5 years old

Cystic fibrosis drug to be reimbursed for children aged 2 to 5 years old
Outgoing Public Health and Social Affairs Minister Frank Vandenbroucke. Credit: Belga/Kristof Van Accom

The drug Kaftrio, for the treatment of cystic fibrosis, will now also be fully reimbursed in Belgium for eligible children between 2 to 5 years old from February 2025, announced outgoing Public Health Minister Frank Vandenbroucke.

For two years now, Kaftrio has gradually been more widely reimbursed for people with cystic fibrosis: since 1 September 2022, it has been reimbursed for people with the disease over the age of 12 with at least one F508del mutation (more than 800 people). That reimbursement was extended for the first time on 1 February 2023 for children between 6 and 11 years old (about 140 children).

The reimbursement will now be further extended from 1 February 2025, to include children aged 2 to 5 years old who meet the medical criteria. The drug comes in a new form (granules) for these young children. This will allow about 70 children with cystic fibrosis to be treated with the drug.

Clinical trials

"It is absolutely my intention to ensure that the best medicine is available to patients, and so also to improve the lives of people with cystic fibrosis and their families," Vandenbroucke (Vooruit) said.

Vandenbroucke's office also reports that clinical trials for the use of Kaftrio in babies as young as one year old are underway. "Unfortunately, it will take a few more years before we know whether Kaftrio is safe in 1-year-old babies and produces good results."

Cystic fibrosis, also called mucoviscidosis, is a congenital, life-threatening and so far incurable disease. In Belgium, 1,379 people suffer from it. Thanks to scientific research, treatments are improving. New drugs help improve quality of life and increase life expectancy.

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