2023-11-23 09:00:32
In France, around 200 children are born each year with cystic fibrosis and around 6,000 people live with the disease. Several new drugs have been put on the market in recent years, improving the prognosis of patients. There High Authority of Health (HAS) has been working for several months to allow the youngest children to access these treatments, from the age of one. Explanations.
Targeted therapies in cystic fibrosis
In 2010, an era of therapeutic innovations began in the cystic fibrosis, allowing the arrival of several new drugs, directly targeting the protein affected by the genetic mutations. Previously, treatments were only symptomatic. From now on, therapeutic solutions come to compensate the lack of functionality of the CFTR protein (Cystic Fibrosis Transmembrane Conductance Regulator).
Currently, four drugs of this type are available in France:
- Le Kalydeco® (ivacaftor) ;
- Orkambi® (lumacaftor combined with ivacaftor);
- Kaftrio® (ivacaftor combined with tezacaftor and elexacaftor);
- Symkevi® (tézacaftor combined with ivacaftor).
All act on the CFTR protein and thus improve weight gain et respiratory functions of patients.
Treatments accessible following several years with cystic fibrosis
Not all patients can benefit from these treatments, which are only intended for patients with certain genetic mutations. On other genetic mutations, these treatments are not effective. Cystic fibrosis is in fact a genetic disease, caused by different mutations affecting the gene coding for the CFTR protein. Another criterion that can be limiting in benefiting from these new treatments is the age of the patients. When they are put on the market, new drugs are aimed at patients who are at least a few years old. From now on, HAS recommends opening access to these treatments to younger patients, sometimes from the age of one.
Initially, the drugs Kaftrio® and Kalydeco® were indicated for patients aged 6 years and older. In September 2023, HAS granted an early access decision to these two medications for children aged 2 to 5 years. Thanks to this decision, around 500 children became eligible for these new cystic fibrosis treatments. And the HAS wants to go even further. At the end of October 2023, she declared herself in favor of early access to the drug Orkambi® from the age of one.
Allow children to be treated from the age of one
This new decision will make it possible to prescribe the drug Orkambi® to children carrying two F508del mutations from the age of one. Previously, this treatment was only available to children ages 2 and older. Thanks to this new early access decision, around 50 children will be able to access this innovative treatment from the age of 1. Such an early access decision might occur in the coming months for the drug Kaftrio® to benefit children from the age of one.
These decisions of the HAS now make it possible to prescribe specific treatments to children suffering from cystic fibrosis from a very young age, without waiting several years. For young patients and their families, these new treatments offer hope for a better quality of life and a better prognosis of the disease. They make it possible, from the age of one year, to act directly on the progression of the disease. A major breakthrough in the fight once morest cystic fibrosis.
Estelle B., Doctor of Pharmacy
Sources
– Kaftrio®: More than 500 children aged 2 to 5 will be able to benefit from it as part of early access! Defeat Muco. September 11, 2023. www.vaincrelamuco.org. Accessed November 13, 2023.
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