Eli Lilly headquarters in Indianapolis, Indiana, US, on Wednesday, May 3, 2023. Shares of Eli Lilly & Co. rose in early U.S. trading after the experimental Alzheimer’s drug slowed disease progression in a late-stage trial, clearing the way for the company to file for U.S. approval.
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Eli Lilly said Friday that the Food and Drug Administration has pushed back The approval deadline for the drugmaker’s experimental Alzheimer’s treatment donanemab in a surprise move.
The agency plans to convene a last-minute meeting of its outside advisers to further review the safety and effectiveness of the treatment in a late-stage trial, Eli Lilly said. The FDA has not disclosed the date of that meeting, so a possible approval will likely come after this month.
The FDA was expected to decide whether to greenlight the drug by the end of the first quarter. That deadline was already delayed from the expected approval last year.
Eli Lilly’s drug significantly slowed the progression of Alzheimer’s in a last stage test. But the treatment, along with similar drugs, carries safety concerns related to brain swelling and bleeding.
The agency’s decision to call for an advisory meeting reflects the high stakes of developing treatments for Alzheimer’s disease. The condition affects more than 6 million Americans and currently has no cure, leaving patients with it with few effective care options.
It’s another setback for Eli Lilly, which is struggling to compete Biogen and Eisai. Their Leqembi treatment won approval last year, becoming the first drug shown to slow the progression of Alzheimer’s in people in the early stages of the memory-robbing disease.
Both Leqembi’s and Eli Lilly’s drugs are monoclonal antibodies that target the build-up of a protein in the brain called amyloid plaque, which is considered a hallmark of the disease.
Eli Lilly called the delay “unexpected” but said it is confident in donanemab’s “potential to provide very significant benefits to people with early symptomatic Alzheimer’s disease,” according to a statement.
“We will work with the FDA and stakeholders in the community to make this presentation and answer all questions,” Anne White, president of neuroscience at Eli Lilly, said in a statement.
Eli Lilly noted that while it is unusual for the FDA to hold an advisory panel after a set action date, the agency has convened similar meetings for two other amyloid plaque-targeting therapies it previously approved.
The FDA often turns to an advisory group for advice on whether an unapproved product is safe and effective.
The organization usually follows the recommendations of its advisors, but is not required to. In 2021, the FDA approved an older, ill-fated Alzheimer’s drug called Aduhelm from Biogen and Eisai, despite a negative recommendation from the agency’s advisory panel.
The FDA will review an 18-month phase 3 trial that followed more than 1,700 patients in the early stages of Alzheimer’s who had a confirmed presence of amyloid plaque. The agency is interested in understanding safety outcomes and how the “unique” trial design affected efficacy.
Eli Lilly’s study allowed patients to stop taking the drug once amyloid plaques were shown to be cleared from the brain. Alzheimer’s drugs, including Leqembi, do not have established stopping points for patients.
Eli Lilly’s drug showed positive results in that trial. Patients who received the drug experienced a 35 percent slower decline in their memory, thinking and ability to perform daily activities compared to those who did not receive the treatment, the data showed.
However, 37% of people who received donanemab had brain swelling or bleeding, including three who died, according to the trial. This compares with about 15% of people who received a placebo.
These side effects have also been seen in Leqembi.