A promising treatment for cystic fibrosis


This is a major step forward for people with cystic fibrosis. On Thursday 26 November, the High Authority for Health issued a favorable opinion on the reimbursement of Kaftrio, a very promising treatment that, once marketed, can be prescribed to more than 80% of patients.

Developed by the American laboratory Vertex, this triple therapy does not cure the disease, but acts on the main repercussions: it allows to increase the respiratory capacity of patients, up to 15%, and to reduce digestive disorders. “The observed effects are spectacular. Thanks to this treatment, patients waiting for lung transplantation were removed from the waiting lists. And it gives hope for an improvement in patients’ life expectancy “, Pierre Foucaud, president of the association Vaincre la cysticiscidose, enthuses.

The most common serious and rare genetic disease

Cystic fibrosis is the most common serious and rare genetic disease. It affects around 7,500 people in France. Even today, singer Grégory Lemarchal remains the face of this disease for the general public. He died in 2007 at the age of 23.

“The image of Grégory is still very strong. It proved that even with a poor prognosis you can get to the end of your dreams, says the president of the association Vaincre la cysticiscidose. But some patients were able to experience his death as a sort of countdown to themselves ”.

However, things have changed in recent years. “The median age at death is 34, which clearly indicates the severity of the disease, continues Pierre Foucaud. But research is advancing by leaps and bounds. We can hope that a child born in 2020 can reach at least 50 years “. Currently, the disease affects a small majority of adults (56%).

The breakthrough in protein therapy

Indeed, the progress of research (funded mainly by private laboratories and the Vaincre la cysticoviscidose association) has been very important over the past decade. “The first treatment was developed in 2012 using protein therapy. This corrects and activates the CFTR protein that does not function properly in cystic fibrosis patients ” describes Dominique Hubert, a pulmonologist at the CF Resource and Skills Center (CRCM) at Cochin Hospital.

The effects of this first drug “They were already spectacular at the respiratory level, but it was effective only for some mutations in the gene responsible for cystic fibrosis, that is, only 3% of patients” explains the doctor.

In the United States, treatment costs € 280,000 per patient per year

Treatment with Kaftrio is therefore a turning point because it can be administered to patients with the most common mutation (DeltaF508), i.e. 80% of patients. “We don’t know what this treatment will give in 10 or 20 years, it will be extremely important to continue to monitor patients in real life,” Dominique Hubert tempera. But thanks to the clinical trials that have been carried out, we have already seen a decline of 3 or 4 years which suggests that all the signs are green ”.

This treatment, in the form of two tablets to be taken every day, is already prescribed in France, thanks to a temporary authorization for use, to 200 or 300 patients in critical situations, who no longer have therapeutic alternatives.

The long time to negotiate the price of the drug

In the United States, Kaftrio costs € 280,000 per year for a single patient. The favorable opinion of the Haute Autorité de santé for the reimbursement of the triple therapy opens the way for negotiations between the laboratory and the Economic Committee for Health Products to fix the French price. “It is a particular effort for national solidarity, acknowledges the president of the association Vaincre la cysticiscidose. Negotiations will likely be difficult because France is the European country where drug prices are the lowest. But given the wait for this treatment, we hope these negotiations won’t stall like a previous drug where 4 years have passed between marketing authorization and price setting. During this period, the sick are deprived of it. “

Initially, Kaftrio can only be prescribed to patients over 12 years of age. “This treatment will slow the development of the disease and, if given early enough, patients can have a normal life. Is fantastic ” let go of the pulmonologist who has dedicated her life to patients with cystic fibrosis.


Source link