The Food and Drug Administration-bsp-bb-link> wants to hear from outside experts “to further understand topics related to evaluating the safety and efficacy” of donanemab, Lilly said Friday in a statement. The decision to hold an advisory panel hearing is a major surprise and comes just weeks before the FDA was expected to rule on approving the drug. The meeting date hasn’t yet been set.
The new delay is the latest in a string of setbacks for Lilly’s drug, which removes a toxic protein called amyloid from the brains of Alzheimer’s patients and helped slow cognitive decline-bsp-bb-link> in a large trial. In studies, side effects included brain swelling and brain bleeding, and regular monitoring is required to detect these issues early before they cause problems.
The shares fell 1.6% at 10:26 a.m. in New York after gaining 34% this year through Thursday’s close. The FDA declined to comment, citing rules about possible or pending drug applications or approvals.
Alzheimer’s experts and doctors at a medical conference in Lisbon, Portugal on Friday were generally disheartened and surprised by the news, one attendee at the meeting said.
“It is extremely disappointing to see this decision made at this point when we thought we were so close to seeing this antibody receive approval,” Donna Wilcock, director of the Center for Neurodegenerative Disorders at Indiana University School of Medicine, said in an email.
Wilcock said she doesn’t know why the FDA decided to convene the panel, but that she “can imagine” that the potential for repeat brain scans in patients may be discussed. Lilly’s biggest trial of donanemab allowed some people to stop taking the drug before the trial was over if the scans showed their brains were free of amyloid.
If the drug is approved, that raises the question what an FDA label might say about the possibility of stopping the drug, and when. The challenge for the advisory panel will be how to convert the approach Lilly’s trial used for halting the drug into prescribing recommendations for physicians, Bloomberg Intelligence analyst Sam Fazeli-bsp-person> said.
The sudden FDA move for a new hearing could be a sign of increased caution on complicated drug applications after a firestorm of criticism following the approval of the first amyloid-lowering drug,
Aduhelm was a commercial failure as the federal Medicare program for the elderly largely refused to cover it. In January, Biogen said it planned to stop selling the drug and return rights to the drug to the private company that invented it.
In an interview, Lilly executives said the FDA’s last-minute move to convene the external committee would likely delay a decision on donanemab for a few months, as it will take the agency time to organize and schedule the meeting.
Unique Aspects
While advisory panel hearings are common before major drug approvals, Lilly called the decision to hold the hearing at such a late stage “unexpected” and “unusual.” Company executives were confident that the drug offered clear benefits to patients.
“We just found out very, very recently,” that the agency had decided to hold the hearing, said Anne White, a company executive vice president and president of Lilly Neuroscience.
Among other things, she said, the agency wants to explore “unique aspects” of Lilly’s main donanemab trial, including the fact that some patients are able to stop taking the drug after all the amyloid is cleared from their brains, as well as unspecified topics related to safety. The agency indicated it wants to move quickly to organize the hearing, she said.
If Lilly’s donanemab is approved, it would compete with Eisai Co.-bsp-bb-link>’s Leqembi, which received full FDA approval-bsp-bb-link> in July and is sold with Biogen. It’s the first drug that unambiguously slowed the disease in a large trial — and also went before an advisory panel hearing prior to its approval. Biogen shares rose as much as 4.1% in New York, their biggest intraday gain since Dec. 11.
Review Extension
The planned hearing “increases the likelihood” that the Lilly drug will get a more complicated label than Leqembi, RBC Capital Markets analyst Brian Abrahams-bsp-person> wrote in a note.
Lilly initially applied for accelerated approval for donanemab based on mid-stage results showing it lowered brain amyloid levels. But in January 2023, the FDA refused to grant-bsp-bb-link> the conditional approval and asked Lilly to submit a traditional drug application with data from the bigger, final-stage trial, which Lilly did last year.
The FDA then asked for an extension on reviewing this additional data, pushing off a final decision until early this year, Lilly executives said-bsp-bb-link> in November. Before this latest delay, Lilly repeatedly said that a final decision would come by the end of the first quarter.
As recently as two days ago, Lilly executives insisted everything with the review was proceeding normally. “It’s a completely normal experience so far,” Andrew Adams, a senior vice president, said Wednesday at a TD Cowen investor conference. “As far as I’m aware, everything is completely normal, yes. You’d have to ask the agency for their view, but from our side, everything’s completely normal.”
(Updates with FDA response in fourth paragraph, expert comments in sixth)
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Madison Muller-bsp-person> in New York at mmuller84@bloomberg.net
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Peter Vercoe, John Lauerman
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