Cystic fibrosis: towards universal innovative treatment for children

It should be available in the coming days. The innovative treatment for cystic fibrosis will be extended to children in France, whereas until now it has only been available to patients aged 12 and over, Health Minister François Braun said.

“The decree is ready and should be published in the coming days: Kaftrio, still an experimental treatment, will be extended to children with cystic fibrosis,” the minister said in an interview with the Journal du Dimanche published on Saturday.

“The results are extraordinary, they allow you to lead an almost ordinary life,” he also said. The minister specified that Kaftrio will be “available in pharmacies very quickly, on prescription from the hospital.”

A revolution for patients

Caftrio, produced by the American laboratory Vertex, is part of an innovative category of drugs against this genetic disease, which inexorably impairs the functioning of the respiratory and digestive systems and was once often fatal for children and adolescents.

The treatment is seen by the associations as a revolution, capable of turning cystic fibrosis into a chronic and stabilized pathology for some patients.

Supplied in the form of pills for life, this triple therapy (a combination of three molecules) clearly reduces the consequences of the disease, in particular, especially disabling lung diseases.

The registration certificate was issued in August 2020 in the European Union. From June 2021 in France, people with cystic fibrosis aged 12 years or older who carry a specific mutation may benefit from it, but not yet children aged 6 to 11 years.

Relief patients

The announcement was greeted with “a sense of relief,” David Fiant, president of the association Vaincre la Mucoviscidose, said in a press release Saturday.

In France, “thanks to this renewal of the marketing authorization, more than 700 new patients will be able to benefit from this innovative therapy,” the association points out: in total, during the first quarter of 2023, “that will be about 5,000 people with cystic diseases.” fibrosis”, which will thus gain access to Kaftrio.

The treatment addresses the underlying causes of the disease by repairing defects in the CFTR protein caused by the genetic mutation. However, it is not suitable for all patients. “Nearly 35% of CF patients are still waiting for therapeutic innovations that will enable them to improve the quality of their daily lives,” recalls Vaincre la Mucoviscidose.