Two new innovative treatments for patients with cystic fibrosis will be reimbursed by Social Security. A “relief” because one of them constitutes “a real therapeutic revolution”, according to two associations.
“An agreement on the price has just been reached for the two pharmaceutical specialties Kaftrio and Symkevi” from the American laboratory Vertex Pharmaceuticals, “allowing these treatments to be covered 100% by Health Insurance”, indicates the Ministry of Health on June 28, 2021 in a press release. Two new innovative treatments for patients with cystic fibrosis will thus be reimbursed by Social Security. “The reimbursement of these drugs will take place in the next few days”, he adds. These medicines, together with another treatment, Kalydeco, may be used in children over 12 years old, adolescents and adults.
A real relief
This decision represents “a real relief”, reacted in a press release Vaincre la cysticiscidose and the association Grégory Lemarchal, who launched a petition a few days ago “to market the Kaftrio treatment as quickly as possible in France”. “The hope of one day making cystic fibrosis a stabilized chronic disease is well founded”, commented Pierre Foucaud, president of Vaincre la Mucoviscidose. “Kaftrio, in particular, constitutes a real therapeutic revolution thanks to its spectacular and lasting action for eligible patients (by their genetic mutations), and a good tolerance profile”, explain associations.
Lung transplants
Cystic fibrosis, a rare and incurable genetic disease, causes abnormally thick mucus to secrete, which obstructs the airways and digestive system. It was once often fatal in children and adolescents, but life expectancy has gradually increased thanks to treatment. The current treatments are however very heavy: respiratory physiotherapy, aerosols, drugs … Many patients suffer from pulmonary infections because of the obstruction of the bronchi and must be hospitalized several times a year. Lung transplants can lengthen life.
20 years of research
“Rather than acting on the symptoms”, Kaftrio and Symkevi act on the “underlying causes of the disease” by repairing defects in a protein, CFTR, caused by a genetic mutation, the lab explains. “This medical breakthrough is the result of more than 20 years of research and development efforts”, underlines in a press release Nicolas Renard, CEO of Vertex France. Symkevi received marketing authorization in the European Union in October 2018 and the High Authority for Health (HAS) issued a favorable opinion for its reimbursement in May 2020, judging that it allowed an improvement. “moderate” actual benefit, in association with Kalydeco. Kaftrio, already marketed in the United States under the name Trikafta since October 2019, obtained the green light from the EU in August 2020 and the HAS estimated in November “important” improvement of actual benefit, in association with Kalydeco.
1,200 days of waiting
“It was therefore necessary to wait more than 900 days and 300 days from the marketing authorization (MA) in Europe, respectively for Symkevi and Kaftrio, for these drugs to finally be available in France, while they were already in a dozen European countries “, deplore the two associations. “Over the past months, nearly 500 patients have benefited from it due to the very severity of their respiratory failure”, thanks to a ‘compassionate access procedure’, they specify.
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