Only reserved for over 12 years old, the Kaftrio® – treatment against cystic fibrosis – will be generalized to all children, announced the Minister of Health François Braun in the columns of the Sunday newspaper. “We are working to bring them to market much faster, as we are going to do against cystic fibrosishe said. The decree is ready and should be published in the coming days.”
Available in France for two years, Kaftrio® treatment allows patients to live almost normally. “The results are extraordinary, they allow an almost ordinary life. It is an immense hope for the 5,500 sick children. It will be available in pharmacies very quickly, on hospital prescription.added the Minister. If it does not cure the disease, the treatment given in the form of tablets for life – a combination of three molecules – drastically reduces the effects of the disease responsible for a deterioration of the respiratory and digestive systems, in particular the lung conditions which can be particularly troublesome and avoids a severe course of the disease, sometimes fatal in some children and adolescents.
This extension “for patients aged 6 to 11 with at least one F508del mutation” had been authorized by the European Medicines Agency in January 2022, before being validated by the HAS in March. In May 2022, 12-year-old patients who did not carry the F508del mutation were also able to benefit from this treatment.
More than 5,000 patients will benefit from the treatment
Thanks to the extension of treatment to children under 12, “more than 700 new patients will be able to benefit from this innovative therapy”indicates the association Overcoming cystic fibrosis in a communicated. In total, nearly 5,000 people will thus be able to have access to this innovative treatment: cystic fibrosis affects nearly 7,300 people in France.
“From now on, as soon as the decree is published in the Official Journal, patients aged 6 and over, carriers of at least one F508del mutation, regardless of the second mutation, will be able to be prescribed this new disease-modifying treatment by their CRCM and order it from a town pharmacy”adds the association.
However, this promising treatment does not work in all patients. “Nearly 35% of patients with cystic fibrosis are still waiting for a therapeutic innovation, allowing them to improve their daily quality of life”recalls Overcoming cystic fibrosis.
Sources:
- Cystic fibrosis: Kaftrio, an experimental treatment for children, will be generalized, The Sunday JournalDecember 17, 2022
- Kaftrio: Santa Claus does exist for children, Overcoming cystic fibrosis, December 17, 2022
