Young patients could see their daily lives upset by this announcement. An innovative treatment against cystic fibrosis will be generalized in France for children, whereas it was until now only available for patients aged twelve and over, announced the Minister of Health François Braun. In an interview given to Sunday newspaperpublished Saturday, December 17, he indicated that the decree was ready and should “appear in the coming days”.
“The results are extraordinary, they allow an almost ordinary life”, he also indicated. The Minister specified that the Kaftrio will be “available in pharmacies very quickly, on hospital prescription”. On Twitter, François Braun welcomed this decision and published a letter written by “little Elsa”, a child with cystic fibrosis. Kaftrio, produced by the American laboratory Vertex, is part of an innovative category of drugs against this disease of genetic origin. As a reminder, cystic fibrosis causes abnormally thick mucus to be secreted, inexorably deteriorating the respiratory and digestive systems. It was once often fatal to children and adolescents.
Delivered in the form of tablets for life, this triple therapy (a combination of three molecules) significantly reduces the effects of the disease, in particular particularly disabling lung conditions. Rather than acting on the symptoms, the treatment targets the underlying causes of the disease by repairing defects in a protein, CFTR, caused by a genetic mutation. “The CFTR protein is present in the membrane of cells of different mucous membranes: respiratory, digestive… It functions as a channel which allows the exchange of chloride ions between the inside and the outside of the cell”, details the Institute. national body for health and medical research (Inserm). However, it is not a miracle treatment, because there is still no cure for cystic fibrosis.
A revolution for associations
A marketing authorization for Kaftrio 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 – it was reimbursed – but not yet children aged 6 to 11, which has been the subject of of a long fight on the part of the associations. Unsurprisingly, the generalization of its access was greeted with “a feeling of relief”, said David Fiant, president of the association, in a press release. Defeat Cystic Fibrosis. He was one of the first patients to test this treatment.
The association considers Kaftrio as a revolution capable, for some patients, of transforming cystic fibrosis into a chronic and stabilized pathology. In Youtube videoDavid Fliant announces with Pierre Lemarchal, president of the Gregory LeMarchal association, “this great news” and speaks of “a new step taken”. 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 have access to it.
It should be noted that 200 children with the disease are born each year in France, according to the Inserm website, i.e. on average “1 in 4,500 newborns”, which makes it one of the most common lethal genetic diseases. . David Fliant insists the treatment has shown “very dramatic effects” in those over 12 and studies show that “for 6-11 year olds it’s the same”. “Santa Claus does exist for children!”, tweeted the association Vaincre la Mucoviscidose.
However, the treatment is not suitable for all patients. “Nearly 35% of patients with cystic fibrosis are still waiting for a therapeutic innovation that will allow them to improve their quality of life on a daily basis”, recalls Vaincre la Mucoviscidose. And some 900 patients who have received a transplant cannot claim it either. From now on, the life expectancy of people with cystic fibrosis is over 40 years, whereas it was only 5 years in 1960. And it should still progress.