European regulators have maintained their positive stance on the Alzheimer’s treatment Leqembi, developed jointly by Eisai and Biogen, following a supplementary safety evaluation. This development paves the way for potential authorization by the European Commission.
The European Medicines Agency’s renewed endorsement comes after the European Commission requested an examination of additional safety information that emerged subsequent to the drug’s initial
recommendation in November. After conducting this review, the regulatory committee stood firm in its original position, choosing not to alter its previous assessment.
This advancement represents a significant milestone for Leqembi in Europe, particularly noteworthy given the drug’s initial rejection by the EMA before the companies’ successful appeal led to a reversal of that decision. If approved, the medication would become available across the 30 nations comprising the European Economic Area, where approximately 22 million individuals suffer from Alzheimer’s-related conditions and dementia.
Leqembi, along with its competitor Kisunla from Eli Lilly, functions by eliminating amyloid beta, a harmful protein found in the brains of Alzheimer’s patients. While both medications demonstrate modest success in slowing disease progression, they carry the risk of ARIA (amyloid-related imaging abnormalities), which can manifest as brain swelling or microbleeding.
The presence of these ARIA-related complications has prompted healthcare providers to exercise caution in patient selection for both Leqembi and Kisunla treatments. This concern was initially so significant that it led to the EMA’s rejection of Leqembi in July, with regulators initially concluding that the drug’s cognitive benefits didn’t justify its potential risks.
However, Eisai and Biogen challenged this decision through an appeals process, a pathway that historically rarely results in reversed decisions. The companies achieved an unexpected victory when the panel revised its position after analyzing genetic risk factors. The review revealed that patients with either no copies or a single copy of the ApoE4 gene variant faced lower risk levels, making them suitable candidates for treatment. Conversely, individuals carrying two copies of this gene variant, who typically experience earlier disease onset, were deemed to face excessive risk levels that precluded treatment recommendation.
The progression toward potential European approval marks a remarkable turnaround for Leqembi, especially considering its initial regulatory hurdles. The treatment’s journey through the European approval process highlights the complex balance between therapeutic benefits and safety considerations in Alzheimer’s treatment development.
This development holds particular significance given the substantial population affected by Alzheimer’s across Europe. The potential authorization would provide a new treatment option for eligible patients within the European Economic Area, though careful patient selection based on genetic factors would remain crucial for safe implementation.
The regulatory committee’s sustained positive opinion, even after examining new safety data, suggests confidence in the drug’s risk-benefit profile for appropriate patient populations. This careful consideration of genetic factors in determining treatment suitability represents an important step toward more personalized approaches in Alzheimer’s therapy.