On December 18, François Braun, Minister of Health and Prevention, announced that Kaftrio, an innovative treatment against cystic fibrosis (a genetic disease that can affect several organs including the lungs), is now extended to all children from 6 to 11 years old carriers of a particular mutation.
- Cystic fibrosis is a genetic pathology due to the alteration of CFTR management, according to Inserm.
- Each year, there are approximately 200 births of children affected by cystic fibrosis.
In France, nearly 6,000 patients are affected by cystic fibrosis, according to the National Institute of Health and Medical Research (Inserm). This serious genetic disease is mainly characterized by digestive and respiratory damage. There is no treatment that can cure cystic fibrosis, but an innovative drug could improve the lives of young patients: Kaftrio.
Kaftrio attenuates pulmonary damage linked to cystic fibrosis
In an interview given to Sunday newspaper (JDD), François Braun, Minister of Health and Prevention, announced that the Kaftrio is now open to children aged 6 to 11. “The results are extraordinary, they allow an almost ordinary life”, he pointed out. The drug is available in pharmacies on hospital prescription. Previously, this treatment was only reserved for patients over 12 years old and it was being tested in children under 12 years old.
Kaftrio is based on taking two tablets a day for life. Manufactured by the American laboratory Vertex, this drug considerably improves the daily lives of patients by reducing the effects of cystic fibrosis, in particular lung diseases.
Kaftrio: new hope for improving life expectancy
Asked by European 1Pierre Foucaud, vice-president of the Association “Vaincre la mucoviscidose” affirmed that the results of Kaftrio are impressive in adults. “The first patients were patients who were going to be transplanted with both their lungs because they were in a situation of therapeutic impasse. And the number of lung transplants fell for cystic fibrosis, from 20 to 2 per quarter”he assured.
In a statementthe Association “Vaincre la mucoviscidose” also indicated that the Kaftrio treatment will benefit 700 young patients. “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 doctor and order it from a town pharmacy”can we read in the document.
For now, the life expectancy of a patient affected by cystic fibrosis is over 40 years. But thanks to medical advances, it should still progress. The Association “Vaincre la mucoviscidose” hopes that the Kaftrio will have “spectacular long-term effects” with young children. “More specifically, the hope of seeing their life expectancy radically transformed”, explained Pierre Foucaud, to European 1.
