"The decision is incomprehensible"

The European Medicines Agency (EMA) has recommended the refusal of the marketing authorization for the novel Alzheimer’s drug “Lecanemab” (brand name: Leqembi). EMA considered that the benefits of treatment are not large enough to outweigh the risks.

Although DZNE experts are aware of the potential risks of the drug that may occur in certain cases, they expressly regret the EMA's decision:

"Alzheimer's is a global pandemic and the recommendation for approval of lecanemab would have been an important step towards getting this serious disease under better control in Europe too. The EMA's decision not to recommend approval is incomprehensible, also in view of the fact that Lecanemab has already been approved in other countries," says Prof. Dr. Dr. Pierluigi Nicotera, Scientific Director and Chairman of the Executive Board of DZNE. "In the USA, Japan and other countries that have agreed to the marketing authorization, the urgent, previously unmet clinical need of patients for new treatment options is clearly paramount. Lecanemab is efficacious when administered in very early stages of Alzheimer’s.” European patients, however, who are now in the ideal window of time for treatment with this drug, are now missing out on this therapeutic opportunity, Nicotera says.

"Many patients, their relatives as well as experts had been waiting for Lecanemab," states Prof. Dr. Gabor Petzold, Director of Clinical Research at DZNE and neurologist at University Hospital Bonn. He says the refusal of the drug dashes the hopes of many patients and their families who are dependent on novel therapies for Alzheimer's. "We are losing the chance of a valuable treatment option that is effective in the early stages of the disease. The drug can have side effects. However, these can be identified early by examinations that identify patients who are eligible for treatment, and are also manageable in most patients through regular check-ups of the brain using magnetic resonance imaging," explains Petzold. "Europe is taking a special path here and is falling behind in clinical research if no experience can now be gained in the medical use of this agent."

Prof. Dr. Johannes Levin, Deputy Head of Clinical Research at the DZNE Munich and neurologist at LMU Klinikum Munich, adds: "The refusal to approve Lecanemab is an unexpected setback, as this form of therapy is the first to directly target the causes of the disease and it represents a real breakthrough despite its limited benefits." In addition, if approval had been granted, it would have been possible to initially focus the treatment on patient groups in which a particularly favorable efficacy-side-effect ratio of Lecanemab could be expected, says Levin. "This discussion cannot be held now." The decision could also create the risk of two-tier healthcare, as wealthy patients in Europe could buy the drug from international pharmacies and receive treatment at their own expense, according to Levin.

Prof. Dr. Frank Jessen, member of the DZNE's Clinical Executive Board and psychiatrist at the University Hospital Cologne, explicates: "The countries in which Lecanemab has been approved, such as the USA, have developed detailed recommendations for information and treatment, including criteria for inclusion and exclusion and guidelines for monitoring side effects. This enables patients, together with their relatives and their physician, to make an informed individual decision for or against treatment. The EMA's recommendation, on the other hand, fundamentally deprives all patients in the EU of the opportunity to receive this treatment."

Prof. Dr. Dr. h.c. Christian Haass, speaker of the Munich site of DZNE and biochemist at Ludwig-Maximilians-Universität München, says: "As a scientist who has been researching the role of amyloid proteins in the development of Alzheimer's disease for decades, I am personally shocked by the EMA's decision. Our research and that of many colleagues has shown that harmful amyloid deposits in the brain, so-called plaques, play a central role in Alzheimer's disease and offer an important starting point for novel therapies. Lecanemab directly targets the causes of the disease by helping to break down the plaques in the brain. However, this form of therapy combating amyloid deposits not only reduces the plaques, but also other pathological changes such as the accumulation of another protein called tau in the brain and the death of neurons.” This is accompanied by an improvement in patients' daily activities of up to 38 percent, enabling them to cope better with everyday life, explains Haass. "The refusal of approval finally closes therapeutic windows for countless patients in Europe who would have gained valuable time and quality of life with this immunotherapy."

_{July 2024}