FDA panel makes the call on much-anticipated Alzheimer’s drug donanemab

An advisory panel of the Food and Drug Administration (FDA) endorsed the experimental Alzheimer’s drug donanemab on Monday, citing studies that demonstrated its effectiveness in slowing the early stages of the disease. Despite concerns raised by committee members about potential side effects, particularly brain swelling and bleeds, associated with Eli Lilly’s antibody-based drug, the panel unanimously agreed that the benefits outweighed the risks, particularly for individuals with mild cognitive impairment due to Alzheimer’s disease.

While the FDA is not bound to follow the recommendations of its advisory committees, it typically does so. Notably, the FDA approved Biogen’s amyloid-clearing drug aducanumab in 2021 despite an advisory committee’s recommendation to reject it. However, Biogen later halted sales and relinquished ownership of the drug.

During the FDA’s Peripheral and Central Nervous System Advisory Committee hearing, Eli Lilly officials presented evidence from clinical trials indicating that donanemab effectively slowed cognitive and functional decline in individuals with early-stage Alzheimer’s disease. Despite observed side effects such as brain swelling and amyloid-related imaging abnormalities (ARIA), the panel deemed the drug’s benefits significant.

However, concerns were raised about the higher risk of side effects for patients carrying two copies of the Alzheimer’s susceptibility gene APOE4. While the panel did not vote on specific recommendations for this subgroup, some members suggested they might benefit less from donanemab and urged further study.

Experts emphasized the need for informed discussions between doctors and patients regarding the potential risks and benefits of the drug. They acknowledged that while donanemab is not a cure for Alzheimer’s, it represents a promising option for slowing disease progression, particularly when used in combination with other treatments.

Ultimately, the endorsement of donanemab signals progress in Alzheimer’s treatment, albeit with ongoing considerations for safety and efficacy.