It was previously only available for patients aged 12 and over. But this innovative treatment against cystic fibrosis will now be generalized in France to children, announces the Minister of Health, François Braun. “The decree is ready and should appear in the coming days: Kaftrio, a treatment that has been experimental until now, will be generalized for children with cystic fibrosis,” he said. in an interview at 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”.
For some, the hope of stabilized health
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 the 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 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.
Thousands of patients affected
This announcement was greeted with “a feeling of relief”, said David Fiant, president of the association. Defeat Cystic Fibrosis. 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, “nearly 5,000 people with cystic fibrosis” 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 daily quality of life,” recalls Vaincre la Mucoviscidose.