Alzheimer's: The High Authority for Health refuses accelerated approval of the drug Leqembi

The French National Authority for Health (HAS) ruled on Tuesday, September 9, against an accelerated approval procedure for Leqembi, an anti-Alzheimer's drug that is generating significant expectations, but is far from unanimous regarding its clinical benefits. Lecanemab, marketed under the name Leqembi, shows "modest efficacy (...) associated with a worrying tolerance profile," according to the transparency committee of this independent authority, thus refusing an "early access" procedure for the treatment developed by the American company Biogen and the Japanese company Eisai.

This treatment is the source of considerable medical controversy. Along with another drug with a similar effect, donanemab, marketed under the name Kisunia and developed by Eli Lilly, they are the first in several decades to demonstrate a slight slowdown in the deterioration of the condition of Alzheimer's patients.

Some experts believe these drugs represent a significant step forward in the fight against Alzheimer's disease, the most common form of dementia, with tens of millions of sufferers worldwide. Patient and family associations, however, overwhelmingly support their approval.

But other experts believe the benefits are too modest to make a difference for patients, especially since these treatments are frequently associated with serious side effects, bleeding and brain swelling – which can be fatal.

Leqembi has been authorized across the European Union (EU) since April, but European health authorities initially refused its authorization and subsequently granted it only by restricting its use to the lowest-risk patients and only in the early stages of Alzheimer's disease. In this context, the HAS had to decide on the merits of "early access" to Leqembi in France for patients with early-stage Alzheimer's disease.

This procedure allows for immediate reimbursement of the drug, at the price set by its manufacturer. The health authority, whose opinions are only advisory but generally followed by the government, ultimately decided that nothing justified such emergency approval because it deemed the side effects to be far too serious and frequent in relation to the meager benefits. "The treatment evaluated is not likely to meet an insufficiently covered medical need," the HAS ruled.

This opinion does not, however, exclude the possibility that Leqembi will one day be reimbursed by health insurance. The HAS will decide later on a possible "common law" procedure, which would involve longer and more complex negotiations between the manufacturing laboratories and the health authorities.