European drug regulators have renewed their support for the
Alzheimer’s treatment Leqembi, developed by Eisai and Biogen, following an additional safety assessment. This reaffirmation comes after the European Commission requested a review of newly available safety information that emerged subsequent to the European Medicines Agency (EMA) panel’s initial endorsement in November.
The European Commission is now positioned to make a final approval decision for Leqembi, which could potentially benefit approximately 22 million individuals affected by Alzheimer’s-related disability or dementia across the 30 nations comprising the European Economic Area.
This development marks a significant turnaround for the medication in Europe, considering the EMA’s initial rejection of the drug. The regulatory body reversed its position only after the pharmaceutical companies launched an appeal, a process that typically has a low success rate.
Leqembi, along with its competitor Kisunla from Eli Lilly, functions by eliminating toxic amyloid beta proteins from the brains of Alzheimer’s patients, resulting in a modest slowdown of disease progression. However, both medications 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 to cautious approaches from Alzheimer’s specialists regarding patient selection for both Leqembi and Kisunla treatments. Similar concerns were initially expressed by the EMA panel, which led to their first rejection of Leqembi in July, stating that the drug’s cognitive decline benefits did not adequately balance against its potential adverse effects.
The successful appeal by Eisai and Biogen centered on demonstrating that ARIA risks varied significantly based on patients’ genetic profiles. The panel ultimately concluded that individuals with either no copies or a single copy of the ApoE4 gene variant faced acceptably low risks from the treatment. However, they determined that patients carrying two copies of this gene variant, who typically experience earlier disease onset, faced too high a risk level to justify treatment.
This genetic-based risk stratification proved crucial in changing the regulatory perspective on Leqembi’s safety profile. The distinction between different genetic populations and their corresponding risk levels provided a more nuanced understanding of the drug’s potential benefits and risks, ultimately leading to the positive recommendation.
The latest reaffirmation from European regulators suggests that their confidence in Leqembi’s risk-benefit profile remains stable, even after considering the most recent safety data. This development positions the drug closer to becoming available as a treatment option for eligible Alzheimer’s patients across Europe, representing a significant advancement in the management of this devastating neurological condition.
The progression of Leqembi through Europe’s regulatory system highlights the evolving understanding of Alzheimer’s treatments and the importance of genetic factors in determining patient suitability for specific therapies. It also demonstrates how pharmaceutical companies can successfully navigate regulatory challenges through the presentation of detailed safety data and patient stratification strategies.
As the European Commission approaches its final decision, the potential approval of Leqembi would provide European healthcare providers with an additional tool in their arsenal against Alzheimer’s disease, particularly for patients whose genetic profiles indicate a favorable risk-benefit ratio.