Lilly to file for accelerated FDA approval of Alzheimer's drug after Aduhelm OK opens the floodgates

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Eli Lilly's anticipated filing for its Alzheimer’s disease prospect donanemab seemingly poses another challenge to the FDA. (FDA)

Investors predicted that Eli Lilly would be the real winner from the FDA's approval of Biogen's Aduhelm, and here's your proof. 

Lilly plans to file for accelerated approval of its Alzheimer’s disease prospect donanemab later this year, a therapy the company once thought would need boat loads more data to get over the regulatory finish line. 

The planned filing on the strength of phase 2 data comes hot on the heels of the FDA’s decision to grant accelerated approval to Biogen’s Aduhelm. 

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Lilly CFO Anat Ashkenazi downplayed the prospect of filing for approval on the basis of the midphase trial late last month. Talking at an investor conference, Ashkenazi said Lilly expected to need two controlled studies for an approval, adding that the traditional requirement “should be the case, because then you have more data and ability to see segment information across patients, less variability, et cetera.”

Two weeks after Ashkenazi made those comments, the FDA reset the evidence bar for getting drugs against Alzheimer’s to market by granting accelerated approval to Biogen’s Aduhelm on the strength of a surrogate endpoint. Lilly’s rapid U-turn suggests the FDA may have opened the floodgates to new Alzheimer’s drugs.

Lilly is running a phase 3 clinical trial of donanemab but, with the FDA opening the door to approval of Alzheimer’s treatments without the usual proof of clinical efficacy, the company no longer sees a need to wait for the data before trying to get the product to market. Lilly disclosed news of its plan as part of a statement about the FDA awarding breakthrough-therapy status to donanemab.

The anticipated filing seemingly poses another challenge to the FDA. Ashkenazi said Lilly was in ongoing discussions with the agency, which may have detoured into offering encouragement to the company. The challenge stems from differences between the amount of data on donanemab and Aduhelm.

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While Biogen failed to meet the bar for traditional approval, it tried to generate the required data and in doing so built a safety database featuring more than 3,000 subjects. Lilly has data on less than 500 subjects. 

The data that are available are mixed from an efficacy perspective, with the drug failing to improve dementia compared to placebo, but the precedent set by Aduhelm suggests evidence of a reduction in amyloid beta plaques on PET imaging will be enough for accelerated approval.