The Minister of Health, François Braun, announced the generalization to all children of an innovative treatment against cystic fibrosis, Kaftrio.
Source AFP
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Lhe news should come as a real relief to families of children with cystic fibrosis. An innovative treatment, until now only available to patients aged twelve and over, will be made available to all children. “The decree is ready and should be published in the coming days: Kaftrio, an experimental treatment until now, will be generalized for children with cystic fibrosis”, indicated the Minister of Health, François Braun, in an interview. to Sunday newspaper.
“The results are extraordinary, they allow an almost ordinary life,” he also said. The minister specified that the Kaftrio will be “available in pharmacies very quickly, on hospital prescription”.
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” A revolution “
Kaftrio, produced by the American laboratory Vertex, is part of an innovative category of drugs against this disease of genetic origin, which inexorably deteriorates the respiratory and digestive systems and was once often fatal to children and adolescents. The treatment is considered by associations as a revolution capable, for some patients, of transforming cystic fibrosis into a chronic and stabilized pathology. Delivered in the form of tablets for life, this triple therapy (a combination of three molecules) clearly reduces the effects of the disease, in particular particularly disabling lung conditions.
A marketing authorization was issued in August 2020 in the European Union. Since June 2021, people with cystic fibrosis aged 12 and over and carrying a specific mutation could benefit from it in France, but not yet children aged between 6 and 11 years old.
This announcement was greeted with “a feeling of relief”, said David Fiant, president of the Vaincre la Mucoviscidose association, in a press release on Saturday. In France, “thanks to this marketing authorization extension, more than 700 new patients will be able to benefit from this innovative therapy”, indicates the association: in all, during the first quarter of 2023, “this will be nearly 5,000 people with cystic fibrosis” who will thus have access to Kaftrio.
The treatment acts on the underlying causes of the disease by repairing defects in a protein, CFTR, caused by a genetic mutation. However, it is not suitable for all patients. “Nearly 35% of patients with cystic fibrosis are still waiting for a therapeutic innovation, allowing them to improve their quality of daily life”, recalls Vaincre la Mucoviscidose.