European drug regulators have maintained their favorable position on the Alzheimer’s treatment Leqembi, developed by Eisai and Biogen, following a supplementary safety assessment. This development paves the way for the European Commission to make its final approval decision, according to an announcement from the pharmaceutical companies on Friday.
The European Medicines Agency’s renewed endorsement came after the European Commission requested an evaluation of newly available safety information that emerged subsequent to the EMA panel’s initial positive recommendation in November. After conducting this additional review, the committee stood firm in its original position.
Should approval be granted, Leqembi would become available across the European Economic Area’s 30 member states, where approximately 22 million individuals suffer from Alzheimer’s-related impairments or dementia. This represents a significant turnaround for the drug in Europe, particularly considering the EMA’s initial rejection before reversing its stance following an appeal by the pharmaceutical companies.
Leqembi, along with its competitor Kisunla from Eli Lilly, works by eliminating toxic amyloid beta proteins from the brains of Alzheimer’s patients, resulting in a modest slowdown of disease progression. However, these treatments carry the risk of ARIA (amyloid-related imaging abnormalities), which can manifest as brain swelling or microscopic bleeding.
The occurrence of ARIA-related incidents has led to cautious approaches from Alzheimer’s specialists when prescribing either Leqembi or Kisunla. 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-slowing benefits did not adequately offset its serious side effect risks.
Following this rejection, Eisai and Biogen initiated an appeal process, which typically has a low success rate for reversing such decisions. However, the companies succeeded in their appeal when the panel subsequently determined that ARIA risks were sufficiently low in certain genetic populations to warrant the drug’s use. Specifically, the panel found that individuals with either no copies or a single copy of the ApoE4 gene variant faced acceptable risk levels, while those carrying two copies of the variant – who typically experience earlier disease onset – faced risks deemed too significant.
This latest development marks a crucial step forward in expanding treatment options for Alzheimer’s patients across Europe, though careful consideration of genetic factors will play a key role in determining suitable candidates for the therapy. The progression toward potential European approval underscores the evolving landscape of Alzheimer’s treatment, where the balance between therapeutic benefits and safety concerns continues to shape regulatory decisions and clinical practice.
The journey of Leqembi in Europe illustrates the complex nature of drug approval processes, particularly for treatments targeting challenging conditions like Alzheimer’s disease. The successful appeal and subsequent maintenance of the positive opinion demonstrate how additional data and careful risk-benefit analysis can influence regulatory outcomes, potentially leading to broader access to new therapeutic options for patients in need.