An innovative treatment for cystic fibrosis will now be accessible to young children carrying a specific mutation, a decision that will bring “enormous hope” to families, according to a leading association.
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 previously often fatal for children and adolescents.
A revolution
The treatment is considered by associations as a revolution capable, for certain patients, of transforming cystic fibrosis into a chronic and stabilized pathology.
Last year, its access became generalized for children aged 6 years and older who carry the so-called F508del mutation, relatively common in patients with cystic fibrosis. In an opinion published on Monday, the High Health Authority (HAS) decided to open early access to treatment to patients aged 2 to 5 years who also have this mutation.
And, in children over six years of age, the HAS is also deciding to expand access to other patients: 177 mutations, rarer than F508del, will be affected.
500 children affected
Presented as lifelong tablets, this triple therapy (a combination of three molecules) significantly reduces the effects of the disease, particularly debilitating lung conditions.
“It is an immense hope for these patients and their families to stop the progression of the disease and, in particular, the deterioration of their state of health, particularly at the respiratory level, from a very early age,” the Beat association reacted. cystic fibrosis. According to the association, 500 children are affected by this system.
From now on, almost three quarters (72%) of the population suffering from cystic fibrosis will be able to access triple therapy, that is, about 5,500 patients, it is congratulated.
On the contrary, 28% of patients, that is, about 2,200, continue to be excluded due to their genetic profile, because they are transplant recipients or because they are children under two years of age, Vaincre recalls cystic fibrosis.
Source: BFM TV
