The European Medicines Agency (EMA) has maintained its favorable position on the Alzheimer’s treatment Leqembi, developed jointly by Eisai and Biogen, following an additional safety assessment. This development moves the medication closer to receiving approval from the European Commission for use across the European Economic Area.
The EMA’s renewed endorsement comes after the European Commission requested a review of new safety information that emerged subsequent to the agency’s initial positive recommendation in November. After examining the additional data, the committee decided to stand by its original position, indicating that the drug’s benefits outweigh its potential risks for certain patient populations.
This progress marks a significant turnaround for Leqembi in Europe, where it initially faced rejection before successfully navigating an appeals process. If approved, the medication would become available to patients across 30 European nations, where approximately 22 million individuals suffer from Alzheimer’s-related disabilities or dementia.
Leqembi, along with its competitor Kisunla from Eli Lilly, functions by eliminating amyloid beta, a harmful protein that accumulates in the brains of Alzheimer’s patients. While both medications have
demonstrated the ability to modestly slow 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 led healthcare providers to exercise caution in prescribing these medications. This concern was initially so significant that it prompted the EMA to reject Leqembi in July, stating that its cognitive benefits did not sufficiently offset the risk of serious adverse effects.
However, Eisai and Biogen challenged this decision through an appeal – a process that historically rarely results in a reversal. The companies successfully demonstrated that the risk of ARIA varies significantly based on patients’ genetic profiles. Specifically, individuals with either no copies or a single copy of the ApoE4 gene variant face lower risks, making them suitable candidates for treatment. Conversely, patients carrying two copies of this gene variant, who typically experience earlier disease onset, were deemed to face excessive risks that precluded treatment recommendation.
This genetic differentiation proved crucial in changing the regulatory perspective on Leqembi’s risk-benefit profile. The ability to identify patients who are less susceptible to ARIA complications through genetic testing has provided a pathway for safer, more targeted use of the medication.
The drug’s potential approval in Europe would represent a significant expansion of treatment options for Alzheimer’s patients in the region. It would provide healthcare providers with an additional tool to combat this devastating neurodegenerative condition, particularly for those patients whose genetic profile indicates a lower risk of complications.
The progression toward European approval aligns with the global effort to address the growing challenge of Alzheimer’s disease, which affects millions of people worldwide. The careful consideration of both benefits and risks, particularly through the lens of genetic factors, demonstrates the evolving sophistication of Alzheimer’s treatment approaches.
As the final decision from the European Commission approaches, the medical community and patients alike await the potential introduction of this treatment option in European markets. The authorization would mark another step forward in the ongoing battle against Alzheimer’s disease, while maintaining a balanced approach to patient safety through genetic risk stratification.