FDA to further review study of donanemab for early Alzheimer’s

The FDA will convene an advisory committee meeting to discuss the safety and efficacy of donanemab in early symptomatic Alzheimer's disease, according to the manufacturer.

Eli Lilly & Co. stated in a press release that the FDA’s decision indicates a need for clarification of certain topics related to the novel therapeutic, including safety results in patients treated with donanemab and efficacy implications of the unique trial design, including its limited-duration dosing regimen that allowed patients to complete treatment based on amyloid plaque assessment as well as tau levels as a marker of inclusion.

No firm date for the meeting of the Peripheral and Central Nervous System Drugs Advisory Committee has been set, but according to the release, it will likely take place after the first quarter of this year. The pending meeting follows similar meetings for two other amyloid plaque-targeting therapies the FDA has approved, per the release.

TRAILBLAZER-ALZ 2 is a phase 3, double-blind, placebo-controlled study that enrolled 1,736 older adults with early symptomatic AD (mild cognitive impairment or mild dementia due to the condition) with telltale neuropathology such as presence of tau or amyloid plaque found by positron emission tomography.

Participants of TRAILBLAZER-ALZ 2 were more progressed in their disease compared with participants in similar trials of other amyloid plaque-targeting therapies. Eli Lilly stated in the release that all participants, especially those with earlier disease stages, benefited from treatment with donanemab. Clinical benefits were also evident in participants in a limited-duration regimen.

The key risk associated with donanemab is amyloid-related imaging abnormalities, or ARIA, which can be a life-threatening condition, according to the release.

“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,” Anne White, executive vice president of Eli Lilly & Co. and president of Lilly Neuroscience, said in the release.