Eli Lilly to seek FDA approval for experimental Alzheimer’s drug
pharmafile | June 25, 2021 | News story | Medical Communications | Alzheimer's, Eli Lilly, FDA
Eli Lilly plans to seek accelerated FDA approval for its experimental Alzheimer’s drug donanemab, announcing plans on Thursday to file an application with the agency later this year.
The company has already received Breakthrough Therapy designation from the FDA, which aims to expedite the development and review of drugs that are intended to treat a serious condition.
The decision is a reversal for Lilly, as in April a top company executive told investors that Lilly didn’t think it could submit donanemab for an accelerated approval based on the clinical trial evidence it has accrued. However, earlier this month the FDA controversially cleared Biogen’s Alzheimer’s drug Aduhelm on a conditional basis, citing the therapy’s effect on brain plaques associated with the disease.
The FDA based their Biogen decision on how well the drug removed the sticky clumps of amyloid, a key protein in Alzheimer’s, from the brain. This reduction in amyloid, the FDA said, was “reasonably likely” to translate to slower cognitive decline in patients with the disease.
The approval is highly controversial, however, both because the link between amyloid clearance and clinical benefit remains fiercely debated, and because evidence from two large clinical trials of Aduhelm was contradictory.
One study showed treatment with a high dose led to a roughly 20% slower decline in cognition and function than did placebo. The other did not, raising concerns among many experts that the seeming effectiveness in the first trial could be a false positive result.
Results from the Phase II TRAILBLAZER-ALZ study, published in The New England Journal of Medicine, show Lilly’s drug dramatically reduced amyloid plaque in the brains of trial participants. By 76 weeks, the end of the study, roughly 68% of the 131 patients who had received donanemab were considered negative for brain amyloid.
Researchers also observed slower cognitive and functional decline, as measured by a rating scale, among donanemab-treated patients. The results were encouraging, but some experts questioned the magnitude of benefit.
With accelerated approval, however, Lilly does not need conclusive proof donanemab leads to significant clinical benefit, only that its effects are reasonably likely to predict an eventual improvement. The drug’s clear impact on amyloid, following the FDA’s acceptance of amyloid reduction as a surrogate marker for Aduhelm’s efficacy, could be enough for the agency to agree to review the drug.
On Thursday, following the announcement of Breakthrough Therapy designation, Lilly’s shares rose by 8% in pre-market trading.
Kat Jenkins
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