European regulators have renewed their support for the Alzheimer’s treatment Leqembi, developed by Eisai and Biogen, following an additional safety assessment. This development puts the medication back on course for potential approval across the European Union.
The European Medicines Agency (EMA) maintained its favorable position on the drug after conducting a thorough review of new safety information that emerged subsequent to the initial endorsement in November. The European Commission had specifically requested this supplementary safety evaluation, which ultimately did not alter the agency’s original supportive stance.
Should Leqembi receive final authorization, it would become available across 30 nations within the European Economic Area, where
approximately 22 million individuals suffer from Alzheimer’s-related impairments and dementia. This progress marks a significant turnaround for the drug in Europe, particularly considering the EMA’s initial rejection before reversing its decision following an appeal from the pharmaceutical companies.
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 microhemorrhages.
The presence of these ARIA-related complications has prompted healthcare providers to exercise caution in patient selection for both Leqembi and Kisunla treatments. These safety concerns were initially significant enough for the EMA to reject Leqembi in July, stating that its cognitive decline benefits didn’t sufficiently outweigh the potential adverse effects.
Despite the typically low success rate of appeals in such cases, Eisai and Biogen successfully challenged this decision. The reversal came after the panel concluded that ARIA risks were acceptably low for certain genetic profiles. Specifically, individuals with either no copies or a single copy of the ApoE4 gene variant showed lower risk levels. However, the risk was deemed unacceptable for patients carrying two copies of this gene variant, who typically face earlier disease onset.
This latest regulatory development represents a crucial step forward in expanding treatment options for Alzheimer’s patients across Europe. The companies’ successful navigation of the approval process, particularly their ability to address safety concerns through genetic risk stratification, demonstrates the evolving understanding of personalized medicine in Alzheimer’s treatment.
The acceptance of Leqembi for patients with specific genetic profiles highlights a growing trend toward more targeted therapeutic approaches in neurodegenerative disease management. This strategy allows for better risk-benefit assessment based on individual patient
characteristics, potentially opening new avenues for treating this devastating condition while maintaining appropriate safety standards.
The progression toward potential European approval of Leqembi adds to the global landscape of Alzheimer’s treatment options, where careful consideration of both efficacy and safety continues to shape therapeutic decisions. This development could significantly impact the millions of Europeans affected by Alzheimer’s disease, providing a new treatment option for those who meet the genetic criteria for safer use.
The forthcoming European Commission decision will determine whether this treatment option becomes available to eligible patients across the European Economic Area, potentially marking another milestone in the ongoing effort to combat Alzheimer’s disease while maintaining rigorous safety standards.