Leqembi Back on Track for EU Approval After Safety Review

European regulators have reaffirmed their positive opinion of Eisai and Biogen's Alzheimer's disease drug Leqembi, paving the way for potential approval in the European Union. This development comes after a brief delay to review new safety data, marking a significant milestone in the drug's journey to market in Europe.

EMA Reaffirms Positive Stance Following Safety Evaluation

The European Medicines Agency (EMA) has maintained its endorsement of Leqembi after conducting a thorough review of new safety information. This review was prompted by a request from the European Commission (EC) following the initial positive recommendation by an EMA panel in November.

The decision to uphold the positive opinion clears a major hurdle for Leqembi, bringing it one step closer to approval for use in the 30 countries of the European Economic Area. With an estimated 22 million people in this region affected by Alzheimer's-related disability or dementia, the potential impact of this drug is substantial.

Navigating Efficacy and Safety Concerns

Leqembi, along with Eli Lilly's competing drug Kisunla, represents a new class of Alzheimer's treatments that work by removing toxic amyloid beta protein from the brains of patients. While these drugs have shown promise in modestly slowing disease progression, they also carry risks, particularly the potential for ARIA (amyloid-related imaging abnormalities), which can manifest as micro-bleeding or swelling in the brain.

The EMA's initial rejection of Leqembi in July 2023 stemmed from concerns that the drug's cognitive benefits did not outweigh the risk of serious side effects. However, Eisai and Biogen successfully appealed this decision, leading to a reevaluation of the drug's risk-benefit profile.

Genetic Considerations in Treatment Eligibility

A key factor in the EMA's revised stance is the recognition that the risk of ARIA varies based on a patient's genetic profile. Specifically, individuals with no copies or one copy of the ApoE4 gene variant are considered to be at lower risk for ARIA, making them potentially suitable candidates for Leqembi treatment.

Conversely, patients with two copies of the ApoE4 variant, who are more prone to early-onset Alzheimer's, face a higher risk of ARIA. For this group, the EMA panel deemed the risk too high to justify the use of Leqembi.

This genetic-based approach to patient selection represents a nuanced strategy in balancing the potential benefits of Leqembi against its associated risks, potentially setting a precedent for personalized medicine in Alzheimer's treatment.