New breakthrough Alzheimer's drug gets unanimous backing from FDA advisors
What was the outcome of the FDA advisory committee's vote regarding the approval of donanemab?
The FDA advisory committee voted unanimously in favor of approving donanemab, an experimental Alzheimer's treatment developed by Eli Lilly. All 11 members of the Peripheral and Central Nervous System Drugs Advisory Committee agreed that the evidence from clinical trials supported the effectiveness of the drug for patients with early-stage Alzheimer's disease, and that the benefits of the treatment outweighed the risks6. This positive vote brings donanemab one step closer to receiving final approval from the FDA.
How does donanemab work to treat Alzheimer's disease, according to the clinical trial data?
Donanemab is a monoclonal antibody designed to clear brain amyloid plaque, which is a hallmark of Alzheimer's disease4. According to the clinical trial data, donanemab binds to the N-terminal truncated form of β-amyloid and aids plaque removal through microglial-mediated phagocytosis. In the phase 2 TRAILBLAZER-ALZ trial, donanemab showed significant amyloid plaque clearance and slowed clinical progression of the disease, as measured by the integrated Alzheimer Disease Rating Scale (iADRS). The treatment resulted in clinically meaningful benefits on the iADRS and CDR-SB scales for both the low/medium tau and combined populations.
What are the main risks associated with donanemab as identified during the clinical trials?
The main risks associated with donanemab as identified during the clinical trials are amyloid-related imaging abnormalities (ARIA) and infusion-related reactions. ARIA, which are detected using MRI scans, are most commonly observed as swelling in an area or areas of the brain (ARIA-E) or as brain microbleeds (ARIA-H). While many cases of ARIA are temporary or asymptomatic, ARIA can be serious and even fatal in some cases. In the donanemab treatment group, brain swelling (ARIA-E) occurred in 24% of TRAILBLAZER-ALZ 2 participants. Brain microbleeds (ARIA-H) occurred in 31.4% of participants receiving donanemab, compared to 13.6% of participants on placebo. The majority of ARIA cases were mild to moderate, with 1.6% of participants experiencing serious ARIA.