NEW YORK – Eli Lilly on Friday announced that the US Food and Drug Administration has said it expects to convene a meeting of its Peripheral and Central Nervous System Drugs Advisory Committee to weigh in on the company's application for marketing approval of donanemab, an Alzheimer's disease drug candidate that targets beta-amyloid.
Indianapolis-based Lilly is seeking traditional approval for donanemab as a treatment for early Alzheimer's patients with evidence of beta-amyloid pathology, a hallmark of the neurodegenerative disease, based on data from the Phase III study of the drug, TRAILBLAZER-ALZ 2. The firm submitted a biologics license application in Q2 2023 and previously said it expected to receive a decision from the FDA by 2023 year-end.
The FDA has not yet set a date for the committee meeting but has said it wants feedback on the safety and efficacy of donanemab and the study's limited-duration dosing regimen.
Investigators enrolled more than 1,700 patients into the placebo-controlled TRAILBLAZER-ALZ 2 study based on cognitive assessments, imaging assessment of beta-amyloid, and tau staging by PET scans to identify those with earlier disease.
Patients at earlier stages of disease experienced the strongest results, but all groups of patients benefited from donanemab, regardless of tau level, according to Lilly. The company previously reported that the drug slowed cognitive decline by 22 percent on the integrated Alzheimer's Disease Rating Scale (iADRS) and by 29 percent on the Clinical Dementia Rating-Sum of Boxes (CDR-SB) compared to those on placebo.
"We are confident in donanemab's potential to offer very meaningful benefits to people with early symptomatic Alzheimer's disease," Anne White, Lilly's executive VP and Lilly Neuroscience's president, said in a statement. "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."
The only beta-amyloid-targeting treatment for Alzheimer's that has received full approval from the FDA so far is Eisai and Biogen's Leqembi (lecanemab), for which the FDA also convened a meeting of its Peripheral and Central Nervous System Drugs Advisory Committee. While Eisai and Biogen previously had received accelerated approval from the FDA for another anti-amyloid treatment, Aduhelm (aducanumab), Biogen opted to discontinue development of the controversial drug earlier this year after struggling to market it. Aduhelm will no longer be commercially available beginning Nov. 1.