The two drugs, Kaftrio and Symkevi, act on the causes of the disease: they make it possible to restore the CFTR protein, altered by a genetic modification.
- Both drugs are manufactured by the American laboratory Vertex.
- In France, 5,000 eligible patients could benefit from it.
- In total, more than 7000 people are affected by cystic fibrosis in France.
Two new treatments against cystic fibrosis will be available in the coming days in France. Kaftrio and Symveki will be fully covered by health insurance. The Ministry of Health announces it in a communicated Released Monday, June 28. “An agreement on the price has just been reached for the two pharmaceutical specialties Kaftrio and Symkevi in association with Kalydeco from the Vertex laboratory“, announces the text.
A background treatment
“This agreement on the price of two new pharmaceutical specialties is a tremendous source of hope for patients“, specifies the Minister of Solidarity and Health in the same document. These two drugs can be administered to children over 12 years old, adolescents and adults. They belong to a new category of treatments: they do not act on the symptoms but on the causes of the disease.These molecules work to repair defects in the CFTR protein, caused by a genetic mutation.Their use allows patients to improve their respiratory function and their general health.
Medicines authorized for sale in Europe for several months
Kaftrio and Symveki had obtained marketing authorization from the European Union, in August 2020 for the first and in October 2018 for the second, but France then negotiated the price of these two treatments with the laboratories which manufacture them. The Grégory Lemarchal and Vaincre la Mucoviscidose associations had mobilized to ensure that access to treatment was authorized quickly. The petition had collected more than 38,000 signatures. In publications on Twitter, they are delighted with the agreement reached for the two drugs.
A reimbursement agreement has been reached for #Symkevi® and #Kaftrio®!????
Eventually, 5,000 eligible patients will be able to benefit from it! ????
A huge THANK YOU to @olivierveran and the @VertexPharma and to the 37,458 people who signed the petition! ????@A_GregLemarchalhttps://t.co/k9QjVeCLaT— Defeat Muco (@vaincrelamuco) June 28, 2021
A serious and frequent disease
Cystic fibrosis is a genetic disease, “one of the most common in France” according to Inserm. The alteration of the CFTR gene causes respiratory complications, affecting the lungs, but also has consequences on the digestive and reproductive systems. “This alteration results in particular in a decrease in the water excreted at the level of the mucous membranes and, consequently, in inflammation and thickening of the mucus which covers it.“, specifies Inserm. Each year, around 200 children are born with the disease in France. Their life expectancy is increasing: in the 1960s, it was only five years, today it is 40 to 50. years.
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