Donanemab Decision Delayed by FDA

Eli Lilly and Company (Indianapolis, IN) reported the Food and Drug Administration has delayed a decision regarding the approval of donanemab for the treatment of individuals with Alzheimer disease and will convene an advisory committee meeting to further evaluate the safety and efficacy of this investigational treatment. The advisory committee meeting will discuss the Phase 3 TRAILBLAZER-ALZ 2 (NCT04437511), focusing on safety results in treated patients and the unique trial design of the study.

TRAILBLAZER-ALZ 2 assessed the safety and efficacy of donanemab in individuals aged 60-85 year with early symptomatic Alzheimer disease. The trial enrolled 1,736 participants across 8 countries, focusing on cognitive assessments alongside amyloid plaque and tau imaging. Results showed that all groups benefited from donanemab treatment, with those in earlier disease stages experiencing the most significant improvements. Notably, nearly half of participants completed their treatment within 6 or 12 months. However, a key risk associated with donanemab treatment was amyloid-related imaging abnormalities (ARIA), which can be severe. Other commonly reported risks include infusion-related reactions, headaches, and nausea.

The study demonstrated that donanemab significantly slowed cognitive and functional decline in early Alzheimer disease patients, with a notable reduction in brain amyloid plaque levels within 6 months of treatment initiation.

"We are confident in donanemab's potential to offer very meaningful benefits to people with early symptomatic Alzheimer's disease. It was unexpected to learn the FDA will convene an advisory committee at this stage in the review process, but we look forward to the opportunity to further present the TRAILBLAZER-ALZ 2 results and put donanemab's strong efficacy in the context of safety. We will work with the FDA and the stakeholders in the community to make that presentation and answer all questions," said Anne White, Executive Vice President of Eli Lilly and Company, and President of Lilly Neuroscience.