European regulatory authorities have maintained their positive stance on the Alzheimer’s treatment Leqembi, developed jointly by Eisai and Biogen, following an additional safety assessment. This development clears a crucial hurdle toward the drug’s potential approval in the European Union.
The European Medicines Agency’s renewed endorsement comes after the European Commission requested a review of newly available safety information that emerged subsequent to the initial positive
recommendation made in November. After conducting this supplementary evaluation, the regulatory committee stood firm in its original position, choosing not to alter its favorable outlook on the medication.
Should final approval be granted, Leqembi would become accessible across the European Economic Area’s 30 member nations, where approximately 22 million individuals suffer from Alzheimer’s-related impairments and dementia. This represents a significant turnaround for the drug in Europe, particularly noteworthy given that the EMA had initially rejected the treatment before reversing its decision following an appeal by the pharmaceutical companies.
Leqembi, along with its competitor Kisunla from Eli Lilly, functions by eliminating amyloid beta, a toxic 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 microscopic bleeding.
The prevalence of ARIA-related incidents has prompted healthcare providers to exercise caution in patient selection for these treatments. This concern was initially so significant that it led to the EMA’s rejection of Leqembi in July, with the agency determining that the drug’s cognitive decline 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 key to this reversal was the panel’s recognition that ARIA risks varied significantly based on genetic factors. Specifically, the committee determined that individuals with either no copies or a single copy of the ApoE4 gene variant faced acceptably low risks, making the treatment viable for these populations. However, for patients carrying two copies of this gene variant – who typically experience earlier disease onset – the risk was deemed too substantial to justify treatment.
This progression toward European approval marks a significant milestone in Leqembi’s global expansion and represents an important development in the ongoing battle against Alzheimer’s disease. The decision reflects a careful balancing act between therapeutic benefits and safety considerations, particularly in light of the genetic factors that influence treatment outcomes.
The development also highlights the evolving understanding of personalized medicine in Alzheimer’s treatment, where genetic profiles play a crucial role in determining suitable candidates for therapy. This approach to patient selection, based on genetic markers, represents a more nuanced strategy in the deployment of new
Alzheimer’s treatments, potentially setting a precedent for future therapeutic approvals in this field.
The reaffirmation by European regulators suggests growing confidence in the management of ARIA risks through appropriate patient selection and monitoring protocols. It also indicates a shifting landscape in Alzheimer’s treatment, where the availability of these new therapeutic options is expanding globally, albeit with careful consideration of safety parameters and patient characteristics.