The treatment of cystic fibrosis with Kaftrio, a drug available in France for 1 year, is an unprecedented advance.
- Cystic fibrosis is the most common rare disease in Europe.
- The life expectancy of patients with cystic fibrosis is currently 47 years.
A radical impact on the quality of life of patients: this is the conclusion of the 2021 report of the French registry of cystic fibrosis on the effects of this new drug presented as “exceptional” by associations for the fight against cystic fibrosis.
Cystic fibrosis is a deadly disease
Cystic fibrosis is a genetic pathology where the patient secretes excess mucus and sees his life expectancy greatly reduced.
Kaftrio delivered in the form of tablets is therefore now accessible to a large majority of patients after 12 years (6 years for certain young patients) and constitutes a real therapeutic revolution for patients since it allows them to gain weight, breathe better and have fewer infections.
The Kaftrio could affect 80% of patients without a lung transplant
Indeed, the drug manufactured in the United States is “composed of three molecules which act on a protein that the disease has rendered deficient and which succeed in partially making it work again”, describes Eric Deneuville pediatrician at the Cystic Fibrosis Resource and Competence Center.
Kaftrio is now the first-line treatment in the treatment of cystic fibrosis and must be prescribed to patients immediately indicates the High Authority for Health.
In the future, the drug will probably be given as soon as the diagnosis is made to prevent the disease from damaging the lungs and requiring a transplant, which was to date the only way to prolong the lives of patients.
