The US Food and Drug Administration (FDA) has refused accelerated approval for the investigational anti-amyloid donanemab (Eli Lilly) for early Alzheimer’s disease treatment because of insufficient data, the manufacturer has announced.
The FDA has asked Lilly to provide data from at least 100 patients who received a minimum of 12 months of continued treatment of donanemab, reports Medscape.
The data are part of the Phase 3 TRAILBLAZER-ALZ 2 trial, and most of the results will be available by the second quarter of 2023, said the company.
“While the trial included more than 100 patients treated with donanemab, due to the speed of plaque reduction, many patients were able to stop dosing as early as after six months of treatment, resulting in fewer than 100 patients receiving 12 months of donanemab,” Lilly said.
The FDA also indicated that the data to meet the exposure exception would probably need to include the unblinded controlled safety data from TRAILBLAZER ALZ 2 upon completion.
The delay of donanemab’s fast-tracked approval follows the FDA’s approval earlier this month of the anti-amyloid lecanemab (Leqembi, Eisai) for early Alzheimer’s, based on results from the CLARITY AD trial, which showed that the drug reduced amyloid markers in early AD and resulted in moderately less decline in cognition and function than placebo at 18 months. However, it was also associated with several adverse events.
Dr Howard Fillit, co-founder and chief science officer of the Alzheimer’s Drug Discovery Foundation (ADDF), said donanemab “did what it was intended to do” – remove amyloid plaques in the brain.
“And because of the trial’s innovative design, treatment was halted for patients once the biomarker PET scans confirmed the plaques were gone. In some cases, this occurred in under 12 months, which is why fewer patients stayed on the drug for a full year,” he noted.
“The ADDF applauds the design of the TRAILBLAZER-ALZ trial, which is a mark of great progress in the Alzheimer’s field, and we look forward to reviewing the phase 3 data later this year.”
Medscape article – FDA Says No to Accelerated Approval for Alzheimer’s Drug (Open access)
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