“It’s a relief and a great Christmas present for children,” rejoices Thierry Nouvel, general manager of the association Vaincre la mucoviscidose. On Sunday December 18, the Minister of Health, François Braun, announced to the Sunday newspaper the generalization to children of Kaftrio, an experimental treatment against cystic fibrosis.
Until now reserved for patients aged 12 and over, Kaftrio can now be prescribed for children aged 6 to 11. “The decree is ready and should be published in the coming days, said the minister. The results are extraordinary, they allow an almost ordinary life. It’s a huge hope.” he said, adding that the treatment would be “available in pharmacies very quickly, on hospital prescription”. In total, 5,500 patients (and not children, as François Braun mistakenly stated) will be able to benefit from this treatment, including 700 children, including “200 who have already had early access”, says Thierry Nouvel.
In children, it avoids lung degradation
Authorized in the European Union since 2020, Kaftrio is an innovative treatment for cystic fibrosis, a genetic disease that inexorably deteriorates the respiratory and digestive systems and which, before this discovery, was often fatal. This new treatment, produced by the American laboratory Vertex, is considered by associations as a revolution capable of transforming cystic fibrosis into a chronic and stabilized pathology.
“Kaftrio does not cure but acts on the causes of the disease, by repairing the defects of the CFTR protein caused by a genetic mutation, explains Thierry Nouvel. In adults, it allows a substantial improvement in respiratory capacity and in children it prevents lung deterioration, one of the first symptoms of cystic fibrosis. We do not yet have enough hindsight, but we can think that these capacities will be preserved, whereas before it was generally necessary to resort to a lung transplant. »
Delivered in the form of tablets, this triple therapy (a combination of three molecules) is a lifelong treatment, to be taken twice a day. Its cost is approximately €200,000 per year and per patient, according to the general manager of Vaincre la mucoviscidose. “It is a very large sum for Social Security, and we had to intervene to accelerate its marketing. »
Nearly 35% of patients will not be able to benefit from it
Treatment with “spectacular effects” on lung capacity, Kaftrio does not, however, address the other consequences of cystic fibrosis, such as digestive symptoms in particular, specifies Thierry Nouvel. “But the children’s lives will be completely turned upside down. They will be able to live almost normally. It’s a huge step forward.” he points out.
Unfortunately, not everyone will be able to benefit from it. Kaftrio only acts on one mutation – delta F508 – although there are “more than 2,000”. Although it is the most common, « pAlmost 35% of patients with cystic fibrosis will not be able to access this therapy, recalls Thierry Nouvel. Our association fights for these patients. We fund research to the tune of 4 million euros per year so that they too can one day benefit from a treatment that improves their quality of daily life.”.