“The decree is ready and should appear in the next few days: Kaftrio, a hitherto experimental treatment, will be generalized for children with cystic fibrosis,” the health minister said on Saturday 17 December.
An innovative treatment against cystic fibrosis will be generalized in France for children, whereas until now it was only available to patients aged twelve and over, announced Health Minister François Braun.
“The decree is ready and should appear in the next few days: Kaftrio, a hitherto experimental treatment, will be generalized for children with cystic fibrosis,” the minister said in an interview with the Journal du Dimanche, published this Saturday. December 18th. “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”.
Kaftrio, produced by the American laboratory Vertex, is part of an innovative class of drugs against this disease of genetic origin, which inexorably deteriorates the respiratory and digestive systems and was once often fatal in 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 (combination of three molecules) significantly reduces the effects of disease, especially particularly disabling pulmonary conditions.
More than 700 new patients
The 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 6 to 11.
This announcement was met with “a feeling of relief,” David Fiant, president of the Vaincre la Mucoviscidose association, said Saturday. In France, “thanks to this extension of AD, 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, “almost 5,000 people will be affected by cystic fibrosis diseases ” who will thus 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. “Nearly 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.
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