The High Authority for Health has authorized early access to treatment for young children suffering from a specific cystic fibrosis mutation.
- Cystic fibrosis is a genetic disease that causes damage to the lungs, digestive and reproductive systems.
- The High Health Authority announced early access to Kaftrio, a drug which significantly reduces the effects of cystic fibrosis, to children aged two to five years.
- Around 500 young patients are affected by this early access system.
In a press release published on Monday, September 11, the High Authority for Health (HAS) announced the opening of early access to Kaftrio, a treatment prescribed for cystic fibrosis, to children aged two to five years old with a specific mutation.
Early access to Kaftrio for young patients carrying the mutation F508del
Cystic fibrosis is mainly characterized by damage to the lungs, digestive and reproductive systems. This genetic pathology is due to an anomaly in the gene coding for the CFTR protein, carried by chromosome 7.
Developed by the American laboratory Vertex, Kaftrio is an innovative treatment against this chronic disease. In 2022, the HAS authorized early access to a first treatment for children aged 6 to 11, “but only those who are heterozygous for the F508del mutation in the CFTR gene and carry a mutation in this same gene with minimal function.” As explained the Overcoming Cystic Fibrosis Associationthis treatment is now accessible “to patients with cystic fibrosis aged 2 to 5 years carrying at least one F508del mutation, regardless of the nature of the second mutation and without any severity criteria.
Treatment of cystic fibrosis: 500 children affected
According to the associations, early access to Kaftrio represents “a huge hope” for patients. Nearly 500 patients are affected by this early access system, according to 2021 data from the French Cystic Fibrosis Registry.
This triple therapy (a combination of three molecules), delivered in the form of tablets for life, greatly reduces the repercussions of the disease, particularly pulmonary conditions. Kaftrio will be prescribed by a doctor practicing in the Resource Centers and Cystic Fibrosis Skills (CRCM) and provided by hospital pharmacies. Follow-up will then be supervised by CRCM caregivers.
