European medicines regulators have maintained their favorable stance on the Alzheimer’s treatment Leqembi, developed by Eisai and Biogen, following an additional safety assessment. This development paves the way for potential approval by the European Commission.
The European Medicines Agency’s reaffirmation comes after the European Commission requested a review of new safety information that emerged subsequent to the drug’s initial endorsement by an EMA panel in November. After examining the additional data, the committee stood by its original position.
Should Leqembi receive final approval, it would become available across the 30 nations comprising the European Economic Area, where approximately 22 million individuals suffer from Alzheimer’s-related impairments and dementia. This represents a significant turnaround for the medication 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 these medications show 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 led healthcare providers to exercise caution in prescribing both Leqembi and Kisunla. Similar concerns influenced the EMA panel’s initial rejection of Leqembi in July, when they determined that the drug’s cognitive decline benefits did not outweigh its potential adverse effects.
The manufacturers’ decision to appeal this ruling was notable, as such appeals rarely result in reversed decisions. However, Eisai and Biogen successfully demonstrated 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 acceptable risk levels, while those carrying two copies – who typically experience earlier disease onset – faced risks deemed too substantial.
This genetic risk stratification proved crucial in changing the regulators’ perspective on the drug’s safety profile. The ability to identify patients who might be at lower risk for serious side effects helped establish a more favorable risk-benefit ratio for certain population segments.
The journey of Leqembi in Europe exemplifies the complex nature of drug approval processes, particularly for treatments targeting challenging conditions like Alzheimer’s disease. The careful balancing of therapeutic benefits against safety concerns, along with the consideration of genetic factors that might influence treatment outcomes, highlights the evolving nature of personalized medicine in neurological disorders.
The potential approval would mark a significant milestone in expanding treatment options for Alzheimer’s patients across Europe. This development comes at a crucial time when the continent faces growing challenges in managing its aging population and the associated increase in neurodegenerative disorders.
The reaffirmation by European regulators also represents a validation of the scientific approach to targeting amyloid beta in Alzheimer’s treatment, despite ongoing debates about the effectiveness of this strategy. The decision reflects a growing acceptance of treatments that offer modest but meaningful benefits in a disease area where therapeutic options have been historically limited.