FDA advisors unanimously back Lilly’s Alzheimer's drug

The US FDA declined to grant the pharma giant’s monoclonal antibody (mAb) for Alzheimer’s disease (AD) accelerated approval in January 2023, but on Monday, donanemab edged closer to receiving regulatory authorization.

The agency called on the Peripheral and Central Nervous System Drug Advisory Committee to consider specific aspects of Lilly’s clinical trial prior to donanemab’s Biologics License Application (BLA). The discussion was based on the mAb’s TRAILBLAZER-ALZ II Phase III results.

“The studies demonstrated substantial evidence of efficacy and clinically meaningful slowing of disease progression. The efficacy results were consistent across trials, populations, and endpoints, with greater relative benefit for those treated earlier in their course of disease,” the document stated.

“The program also advances the scientific understanding of amyloid and tau in the clinical course of Alzheimer’s disease and enabled limited duration dosing. The safety profile of donanemab has been well characterized. With appropriate labeling and management, the potential risks are outweighed by the demonstrated benefits on the clinical endpoints in patients with AD.”

Eleven committee members unanimously said the data available showcases the drug’s efficacy and proves the benefits outweigh the risks. However, the group proposed that the drug might need a black-box warning, a label that was introduced to a number of chimeric antigen receptor (CAR) T cell therapies in January. Additionally, they said there is a need to proceed with caution for use of thrombolytic therapy as symptoms of amyloid-related imaging abnormalities (ARIA) because symptoms may be comparable to a stroke.

If the drug is approved, Lilly will join a very small group of companies who have been granted authorization by the FDA for AD treatments. Eisai received accelerated approval for Leqembi (lecanemab) in January 2023, an AD drug that is priced at $26,500 per year, roughly half the original price of Biogen’s drug Aduhelm (aducanumab), which due to efficacy and financing concerns has been withdrawn.

“We are pleased with the committee’s unanimous recognition of donanemab’s positive benefit-risk profile,” Mark Mintun, group vice president of neuroscience research and development for Lilly told us. “We look forward to bringing this treatment option to patients.”