The setback could delay a potential commercial launch of the much-anticipated drug by at least several months if the Food and Drug Administration eventually decides to approve it.
The FDA has issued Lilly a complete response letter for the company’s application for fast-track FDA approval of the drug, called donanemab, Lilly said Thursday.
The FDA has asked Lilly to provide clinical data from at least 100 study participants who were treated with the drug for at least 12 months. Lilly said it previously submitted results to the FDA from a midstage study of donanemab that enrolled fewer than 100 volunteers on the drug for at least 12 months.
Lilly said it would work with the FDA to evaluate the fastest way to get the drug to market. The company is conducting a late-stage study testing donanemab in more patients. Results are expected in the second quarter.
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The FDA said it likely would need data from this larger study to make a decision, Lilly said.
Based on this data, Lilly plans to submit an application to the FDA for traditional approval of donanemab by mid-year.
“We are committed to working with the FDA to ensure this potential drug is delivered to patients in need as quickly as possible,” said Anne White, Lilly’s executive vice president and president of Lilly’s neuroscience business.
The FDA’s rejection of Lilly’s Alzheimer’s drug could hurt the company’s shares, which investors have been piling up over the past few months in part on expectations that the drug could generate multibillion-dollar sales.
A delay in the drug’s possible FDA approval might not have a major impact on Lilly’s financial prospects. Lilly has said that even if it got fast-track approval for the drug in early 2023, initial sales would be modest due to a Medicare policy restricting coverage of new Alzheimer’s drugs.
Alzheimer’s advocates have asked the Centers for Medicare and Medicaid Services to reconsider its policy, but if the agency chose to do so, any change would likely take several months.
SVB Securities analyst David Risinger said in a research note that the FDA’s rejection does not change his opinion on donanemab, as its sales prospects depend on the outcome of the larger, late-stage study, the results of which are expected in the second quarter.
Indianapolis-based company Lilly said it is not changing its financial guidance for 2023.
Lilly had hoped for accelerated FDA approval of donanemab earlier this year. Now, filing a standard drug application mid-year means an FDA decision could be pushed back to 2024 based on typical FDA timelines, which take six to 10 months to review new drug applications.
The FDA sometimes grants accelerated approval to give a drug used to treat a serious medical condition early market entry. The FDA can grant accelerated approval of a new drug based on a more limited body of clinical evidence than standard drug approval.
The agency recently approved another Alzheimer’s therapy. Earlier this month, the FDA granted early approval to a new Alzheimer’s drug from Eisai Co. and Biogen Inc.
The FDA gave the drug, called lecanemab, or Leqembi, conditional approval based on an early study that found it reduced levels of a sticky substance called amyloid in the brains of people with early-stage Alzheimer’s.
In her case, Lilly had applied for accelerated FDA approval of donanemab based on evidence that the drug helped reduce the volume of amyloid, which is thought to be a cause of Alzheimer’s.
The midstage study of more than 250 patients also found that the drug slowed cognitive and functional decline compared to a placebo.
Lilly said many of the subjects in the Midstage study were able to stop taking the drug as early as six months after starting treatment because it quickly cleared amyloid plaque from their brains. As a result, fewer than 100 of these patients were treated in the study for 12 months.
Write to Peter Loftus at [email protected]
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