FDA Postpones Decision on Alzheimer’s Drug; approval was expected this month

The American regulatory agency has decided to convene a group of independent experts for a more detailed evaluation of the effectiveness and safety of the drug

The Food and Drug Administration (FDA) has decided to delay action on a drug for Alzheimer’s, donanemab, which the agency was expected to approve this month. Instead, the FDA will require the donanemab be subjected to scrutiny by a panel of independent experts, the drug’s maker, Eli Lilly, said on Friday.

“The FDA has advised Lilly that it wants to better understand the issues surrounding the evaluation of the safety and efficacy of donanemab, including safety outcomes in patients treated with donanemab and the efficacy implications of the unique study design,” the FDA said. company in a note. communicated.

The decision will likely surprise many Alzheimer’s experts, doctors and patients who expected the drug to come to market soon. The FDA’s decision was surprising for the company, which had expected the drug to be given the green light by the agency in the first quarter of this year.

“We didn’t expect this,” Anne White, Lilly’s executive vice president and president of its neuroscience division, said in an interview. She said that while the FDA often turns to these independent advisory committees when it has questions about drugs, it is unusual to do so “late in the review cycle and beyond the action date that the FDA had provided to us.”

The FDA has not publicly commented on the measure, which will delay any decision on whether to approve donanemab until at least the end of this year. Lilly officials said they expected a few months to pass before the advisory panel held a hearing.

“The FDA has made a commitment to us to act quickly, so we expect them to act soon after the advisory committee,” White said.

The decision to convene an advisory committee reflects the high risks and difficult history of developing treatments for Alzheimer’s. The disease affects more than 6 million Americans, and there are currently no treatments or medications that can restore memory loss or reverse cognitive decline.

For years, the industry has been plagued by failed drug trials. But donanemab, an infusion given once a month, belongs to a new class of drugs that experts hope will help patients by attacking a protein, amyloid, that builds up in plaques in the brains of people with Alzheimer’s.

Last year, the FDA approved another drug in this class, Leqembi, made by Eisai and Biogen. An infusion given every two weeks, Leqembi may somewhat slow cognitive decline in the early stages of Alzheimer’s.

The new drugs are considered only a first step in a potentially fruitful direction, as they may not slow the decline enough to be noticed by patients or families, experts say. The drugs also carry significant safety risks, including swelling and bleeding in the brain.

(The first drug approved in the anti-amyloid class, Aduhelm, was controversial because it had weak evidence; Biogen, the drug’s maker, recently dropped it.)

Donanemab was expected to be approved easily because data showed the drug could also somewhat slow cognitive decline in people with mild symptoms and the safety risks were similar to Leqembi.

Because the study design of donanemab was different from that of Leqembi and included some patients with more complex medical problems, the studies of the two drugs cannot be directly compared.

The donanemab study had two unusual aspects that the FDA indicated it would ask the advisory panel to evaluate, said John Sims, Lilly’s chief medical officer and leader of donanemab’s clinical trials.

One feature would be particularly attractive to patients: Study participants stopped receiving donanemab after their amyloid plaques had cleared to a certain level – about a year for half of the participants who started donanemab – and their decline cognition continued to slow. Lilly scientists estimated that it will take nearly four years for amyloid levels to cross the threshold again.

Sims said he believes the FDA wants to understand more about stopping treatment because “it’s a very unique thing” and that regulators may want to explore whether other anti-amyloid drugs could be stopped at some point.

White said that among doctors and patients, “there’s a lot of enthusiasm for this concept that once you’ve achieved the goal you want to achieve, you don’t need to put patients through additional infusions and visits.”

The other unusual feature of the study involved another protein, tau, which forms tangles in the brain after the buildup of amyloid. Higher levels of tau are more associated with memory and reasoning problems.

The donanemab study divided participants into groups with high tau levels and intermediate tau levels. People with intermediate levels of tau had slower cognitive decline, supporting a widely held theory that treating patients as early as possible in the disease process offers a better chance of relieving symptoms.

Sims said measuring tau was “informative but not necessary to institute therapy for patients, and we had treatment effects across the tau spectrum.” She said the FDA did not indicate “the specifics of what they want to talk about” involving tau, only that it was an issue the advisory committee would consider.

White said, “There are some people here at Lilly who have been working on this project for 35 years, and so you can imagine that it was definitely a disappointment for them to not be able to bring this to patients now.”

But he said the company has confidence in its data and will spend the next few months thinking about “additional analysis we can do to help answer any questions anyone might have.” / This article originally appeared in the New York Times

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Source: Terra

Rose

Rose James is a Gossipify movie and series reviewer known for her in-depth analysis and unique perspective on the latest releases. With a background in film studies, she provides engaging and informative reviews, and keeps readers up to date with industry trends and emerging talents.