The High Authority for Health (HAS) has just authorized access to Kaftrio treatment, against cystic fibrosis, in certain children, aged 6 to 11 years. This treatment was previously reserved in France for those over 12 years old, details AFP, Tuesday March 29, 2022. It will now be reimbursed by Social Security.
A revolutionary treatment
Cystic fibrosis is a disease that causes abnormally thick mucus to secrete, obstructing the airways as well as the digestive system. Kaftrio is part of an innovative class of drugs. It targets the action of the protein at the origin of the disease and is considered by associations as a revolution. According to some patients, this treatment can transform cystic fibrosis into a chronic and stabilized pathology.
Also according to AFP, a HAS commission has “recognized the presumed innovative character of this combination of drugs which, in the long term, would allow patients to breathe better, to be sick less often and to regain weight”.
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About 300 children concerned
Only patients “who are heterozygous for the F508del mutation of the CFTR gene”, whose role is central in cystic fibrosis, explains the HAS, which estimates that less than 300 the number of children concerned, out of a total of more than 7,000 patients of all ages in France.
Read also: Cystic fibrosis. The Kaftrio gives hope to the Virades: “In 24 hours, I no longer coughed”
Kaftrio is a combination of two drugs, developed by the Vertex laboratory: Kalydeco and Kaftrio. This treatment has been approved in France since 2021 for eligible patients over the age of 12, a decision which was hailed as a relief by associations after years of waiting.