INDIANAPOLIS, IN - Eli Lilly and Company (NYSE: LLY) today announced that the U.S. Food and Drug Administration (FDA) expects to convene a meeting of the Peripheral and Central Nervous System Drugs Advisory Committee (PCNS) to discuss the Phase 3 TRAILBLAZER-ALZ 2 trial, which evaluated the efficacy and safety of donanemab in early symptomatic Alzheimer's disease.
The FDA has informed Lilly it wants to further understand topics related to evaluating the safety and efficacy of donanemab, including the safety results in donanemab-treated patients and the efficacy implications of the unique trial design of the TRAILBLAZER-ALZ 2 study, including its limitedduration dosing regimen that allowed patients to complete treatment based on an assessment of amyloid plaque and the inclusion of participants based on tau levels.
The date of the advisory committee meeting for donanemab has yet to be set by the FDA, and, as a result, the timing of expected FDA action on donanemab will be delayed beyond the first quarter of 2024. While it is unusual for an advisory committee to occur after the anticipated FDA action date, the advisory committee meeting for donanemab follows similar meetings for the two other amyloid plaque-targeting therapies the FDA has approved.
"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.
TRAILBLAZER-ALZ 2 is a Phase 3, double-blind, placebo-controlled study to evaluate the safety and efficacy of donanemab in participants ages 60-85 years with early symptomatic Alzheimer's disease (MCI or mild dementia due to Alzheimer's disease) with the presence of confirmed Alzheimer's disease neuropathology. Alzheimer's disease is a progressive and fatal disease that in its early symptomatic stages affects 6-7.5 million Americans. The trial enrolled 1,736 participants, across eight countries, selected based on cognitive assessments in conjunction with amyloid plaque imaging and tau staging by positron emission tomography (PET) imaging.
Compared to participants in similar trials of other amyloid plaque-targeting therapies, the TRAILBLAZER-ALZ 2 participants were more progressed in their disease. All groups of trial participants, regardless of tau level, benefited from treatment with donanemab, with patients in earlier stages of the disease experiencing the strongest results. Donanemab also demonstrated clinical benefits using a limited-duration treatment regimen, with nearly half of clinical trial participants completing their course of treatment in six or 12 months. The key risk associated with donanemab is amyloid related imaging abnormalities, or ARIA, which can be serious and life-threatening. Other most commonly reported risks include infusion-related reactions, headache and nausea.
The Phase 3 TRAILBLAZER-ALZ 2 study results were published in the Journal of the American Medical Association (JAMA). Lilly continues to study donanemab in multiple clinical trials.
