European regulators have reinforced their previous endorsement of Leqembi, an Alzheimer’s treatment developed jointly by Eisai and Biogen, following an additional safety assessment. This development paves the way for the drug’s potential authorization by the European Commission.
The European Medicines Agency’s renewed support came after the European Commission requested a review of newly available safety information that emerged subsequent to the initial positive
recommendation in November. After examining the additional data, the committee maintained its original favorable 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 impairment or dementia. This represents a significant turnaround for the medication in Europe, particularly considering the EMA’s initial rejection of the drug before reversing its decision following an appeal from the pharmaceutical companies.
Both Leqembi and its competitor Kisunla, manufactured by Eli Lilly, function by eliminating a harmful protein called amyloid beta from patients’ brains, resulting in a modest slowdown of Alzheimer’s progression. However, this therapeutic approach carries the risk of ARIA (amyloid-related imaging abnormalities), which can manifest as brain swelling or microscopic bleeding.
The occurrence rate of ARIA-related complications has led to cautious prescribing practices among Alzheimer’s specialists when considering either Leqembi or Kisunla for treatment. Similar concerns influenced the EMA panel’s initial rejection of Leqembi in July, when they determined that the drug’s cognitive decline-slowing benefits did not adequately offset its potential adverse effects.
Despite the typically low success rate of appeals in such cases, Eisai and Biogen successfully challenged the ruling. The panel ultimately concluded that ARIA risks were sufficiently low in certain genetic subgroups to warrant the drug’s use. Specifically, individuals carrying either no copies or a single copy of the ApoE4 gene variant demonstrated acceptable risk levels. However, the risk was deemed excessive for those with two copies of this variant, who typically experience earlier disease onset.
The path to potential European approval has been marked by careful consideration of both the drug’s benefits and risks. The EMA’s decision to uphold its positive opinion after reviewing additional safety data suggests confidence in the treatment’s risk-benefit profile for appropriate patient populations. This development represents a crucial step toward expanding treatment options for Alzheimer’s patients in Europe, particularly given the significant number of individuals affected by the condition across the European Economic Area.
The regulatory journey of Leqembi in Europe highlights the complex balance between providing innovative treatments and ensuring patient safety. The initial rejection, successful appeal, and subsequent safety review demonstrate the thorough evaluation process required for new Alzheimer’s treatments, especially those employing novel mechanisms like amyloid beta clearance.
As the decision now moves to the European Commission, the potential approval of Leqembi could mark a significant advancement in
Alzheimer’s treatment options available to European patients. The careful consideration of genetic factors in determining suitable candidates for the therapy reflects an increasingly personalized approach to Alzheimer’s treatment, where individual patient
characteristics play a crucial role in treatment decisions.