European regulatory authorities have maintained their favorable position on the Alzheimer’s treatment Leqembi, developed jointly by Eisai and Biogen, following a supplementary safety assessment. This development puts the medication back on course for potential approval in the European Union.
The European Commission had previously requested the European Medicines Agency (EMA) to examine additional safety information that became available after an EMA committee’s initial endorsement in November. After conducting this review, the committee stood by its original positive recommendation, choosing not to alter its stance on the drug.
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 dementia or disability. This advancement represents a significant turnaround for the drug in Europe, particularly considering the EMA’s initial rejection before reversing its decision following an appeal by the pharmaceutical companies.
Leqembi, along with its competitor Kisunla from Eli Lilly, functions by eliminating a harmful protein called amyloid beta from patients’ brains, resulting in a modest slowdown of Alzheimer’s progression. However, both medications carry the risk of ARIA (amyloid-related imaging abnormalities), which can manifest as brain swelling or minor bleeding.
The occurrence of ARIA-related incidents has led healthcare providers to exercise caution when prescribing these medications. This concern was initially reflected in the EMA panel’s first rejection of Leqembi in July, when they determined that the drug’s cognitive
decline-slowing benefits did not sufficiently outweigh its potential adverse effects.
In an unusual turn of events, Eisai and Biogen’s appeal of this decision proved successful, despite the typically low success rate of such appeals. The panel ultimately concluded that the ARIA risk was acceptable for patients with specific genetic characteristics. Specifically, individuals with either no copies or a single copy of the ApoE4 gene variant were found to have a lower risk profile. However, the risk was deemed too significant for patients carrying two copies of this gene variant, who typically experience earlier onset of the disease.
The regulatory journey of Leqembi in Europe highlights the complex balance between therapeutic benefits and safety considerations in Alzheimer’s treatment. The evolving understanding of genetic factors in patient response has played a crucial role in shaping the regulatory perspective on the drug’s approval pathway.
The potential authorization of Leqembi would provide European healthcare providers with an additional tool in their arsenal against Alzheimer’s disease, though careful patient selection based on genetic profiles will remain crucial. This development comes at a time when the medical community continues to seek effective treatments for this devastating neurodegenerative condition.
The path to potential European approval for Leqembi demonstrates the evolving nature of drug evaluation processes, where initial rejections can be overcome through additional data analysis and careful consideration of patient subgroups. This approach reflects the growing trend toward more personalized medicine, where genetic factors play an increasingly important role in determining appropriate treatment options.
The decision also underscores the importance of post-initial review safety assessments in the drug approval process, as regulatory bodies work to ensure both the efficacy and safety of new treatments while making them available to patients who could benefit from them.