Eli Lilly and Company (Lilly) announced that FDA had rejected its accelerated approval application for donanemab as a treatment for early symptomatic Alzheimer's disease on Jan. 19, 2022. In its response letter, FDA cited concerns surrounding an insufficient quantity of clinical trial data.
According to a company press release, FDA specifically requested that Lilly provide data from at least 100 patients who received a minimum of 12 months of continued treatment on donanemab. Lilly cited the experimental design of the study, which allowed patients to complete their treatment once targeted deposits of amyloid plaque were reduced to predefined endpoints, as causing the insufficient quantity of data. Lilly is currently in the middle of a confirmatory Phase III clinical trial (TRAILBLAZER-ALZ 2), with topline data expected in the second quarter of 2023.
"We anticipate [TRAILBLAZER-ALZ 2] will confirm the benefit and safety profile we observed in the TRAILBLAZER-ALZ Phase II study and believe that patients and physicians will be well served by having the full Phase III data available alongside our Phase II data when they need to make treatment decisions," said Anne White, executive vice-president and president, Lilly Neuroscience, Eli Lilly and Company, in the press release. "We are committed to working with the FDA to ensure the fastest possible path to bring this potential medicine to patients in need."