From June 2021, people with cystic fibrosis aged 12 and older and carrying a specific mutation could benefit from this treatment in France, but not yet children aged 6 to 11.
It is a hope for thousands of children in France and around the world. Health Minister François Braun, announced Kaftrio’s generalization this Saturday, a cure for cystic fibrosis for the 5,500 affected children due to illness in the country.
The treatment, “until then experimental”, will therefore be “generalized”, thanks to its “extraordinary results”, which “allow an almost ordinary life”. A “great hope” for children affected by this disease which secretes abnormally thick mucus, blocks the airways and digestive tracts, and whose life expectancy for patients is between 40 and 50 years .
“To not have too much damage to my body”
In Titouan, 7 years old, suffering from mucovicisosis, the wait is immense. The boy, who takes 14 capsules a day, can’t wait to start his new treatment.
“I need it to do good for my body so as not to get sick, to get better, to not have too much damage to my body”, he confides into the microphone of BFMTV.
Thanks to Kaftrio, Titouan will be able to breathe better, get sick less often and gain weight back. The first effects are visible from the 15th day of treatment. The treatment in question consists of a combination of two drugs developed by the Vertex laboratory, Kalydeco and Kaftrio.
A new hope for children, but also for parents. “We tell ourselves that they can study, that they will have a future, a family with much more confidence. There is this treatment which is revolutionary and which only opens doors. It will continue so it is fantastic,” explains Marjolaine, Titouan’s mother .
About 700 new patients
A 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 between 6 and 11.
Kaftrio, which is part of an innovative class of drugs aimed at the action of the protein at the origin of the disease, is considered by the associations as a revolution which, for some patients, can transform cystic fibrosis into a chronic and stabilized pathology. The Minister of Health clarified that Kaftrio will be “available in pharmacies very quickly, on hospital prescription”.
However, the treatment is not suitable for all patients. “Almost 35% of patients with cystic fibrosis are still waiting for a therapeutic innovation that allows them to improve their quality of life on a daily basis”, recall AFP and the Vaincre la Mucoviscidose association.
