It was previously only available to patients 12 years of age and older. 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 be published in the next few days: Kaftrio, a hitherto experimental treatment, will be generalized for children with cystic fibrosis,” he indicated. in an interview with Sunday newspaper.
“The results are extraordinary, they allow for an almost ordinary life,” he also said. The minister specified that Kaftrio will be “available in pharmacies in a very short time, on hospital prescription”.
For some, the hope of stabilized health
Kaftrio, produced by the American laboratory Vertex, belongs to an innovative category of drugs against this disease of genetic origin, which inexorably deteriorates the respiratory and digestive systems and was once often fatal for children and adolescents. The treatment is considered by the associations a revolution capable, for some patients, of transforming cystic fibrosis into a chronic and stabilized pathology. Provided in tablet form for life, this triple therapy clearly reduces the effects of disease, especially disabling pulmonary conditions.
A marketing authorization was granted in August 2020 in the European Union. From June 2021, people with cystic fibrosis aged 12 and older and carrying a specific mutation could benefit from it in France, but not yet children aged between 6 and 11.
Thousands of patients affected
This announcement was met with “a feeling of relief,” said David Fiant, president of the association. Defeat Cystic Fibrosis. In France, “thanks to this extension of the marketing authorization, more than 700 new patients will be able to benefit from this innovative therapy”, indicates the association. In all, in the first quarter of 2023, “nearly 5,000 people with cystic fibrosis” will have access to Kaftrio.
The treatment targets 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. “Almost 35% of patients with cystic fibrosis are still waiting for a therapeutic innovation that allows them to improve the quality of their daily life,” recalls Vaincre la Mucoviscidose.
