European medication regulators have renewed their positive assessment of the Alzheimer’s treatment Leqembi, developed jointly by Eisai and Biogen, moving the drug closer to receiving approval in the European Union. This reaffirmation comes after additional safety evaluations were conducted at the European Commission’s request.
The European Medicines Agency (EMA) maintained its favorable stance on the medication following a review of new safety information that emerged after the drug initially received endorsement from an EMA committee in November. The committee’s position remained unchanged after examining the latest data.
This development represents a significant turnaround for Leqembi in Europe, where the EMA had initially rejected the medication before reversing its decision following an appeal by the pharmaceutical companies. Upon approval, the drug would become available across the 30 nations within the European Economic Area, where approximately 22 million individuals suffer from Alzheimer’s-related disabilities or dementia.
Leqembi, along with its competitor Kisunla from Eli Lilly, functions by eliminating toxic amyloid beta proteins from the brains of Alzheimer’s patients, resulting in a modest slowdown of disease progression. However, both medications carry the risk of ARIA (amyloid-related imaging abnormalities), which can cause brain swelling or microscopic bleeding.
The presence of these ARIA-related complications 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 benefits did not outweigh the potential side effects.
Despite the typically low success rate of appeals in such cases, Eisai and Biogen successfully challenged the ruling. The panel ultimately determined that ARIA risks were sufficiently low for certain genetic profiles to warrant the drug’s use. Specifically, individuals with either no copies or a single copy of the ApoE4 gene variant were found to have lower risk levels. However, the panel maintained that the risk remained too high for patients carrying two copies of this gene variant, who typically experience earlier onset of the disease.
The current positive recommendation sets the stage for the European Commission to make its final approval decision, which would mark a significant expansion in the availability of modern Alzheimer’s treatments across Europe. This development is particularly noteworthy given the growing prevalence of Alzheimer’s disease in the region and the limited treatment options currently available.
The journey of Leqembi in Europe illustrates the complex balance regulatory bodies must strike between providing access to innovative treatments and ensuring patient safety. The initial rejection, successful appeal, and subsequent safety review demonstrate the thorough evaluation process new medications must undergo before reaching patients in the European market.
This regulatory progress also highlights the evolving understanding of how genetic factors influence treatment safety and efficacy in Alzheimer’s disease. The recognition that ApoE4 gene status affects ARIA risk has helped establish more precise guidelines for patient selection, potentially allowing for safer implementation of these new treatment options.
For the millions of Europeans affected by Alzheimer’s disease, this regulatory advancement offers hope for expanded access to newer treatment options, while maintaining careful consideration of safety profiles based on individual genetic characteristics.