Patient distress as FDA delays decision to approve Eli Lilly’s ‘life-changing’ Alzheimer’s drug over concerns about deadly side effects and how the drug was tested

An unexpected move by the FDA to delay approval of a potentially life-changing Alzheimer’s drug has disappointed doctors and patients anxiously awaiting its availability.

Eli Lilly, the maker of the drug known as donanemab, said approval would be delayed beyond March as the FDA plans to convene a last-minute meeting of outside experts to review its safety and effectiveness.

The drug was expected to gain approval by the end of the first quarter of the year, after clinical trials showed it was capable of modestly slowing the rate of cognitive decline associated with the disease.

But the FDA wants an independent opinion on the drug’s main side effect: It can cause fatal brain swelling.

The FDA wants outside experts to examine the company’s unique trial design, which allowed patients to stop taking the drug once the plaques believed to cause Alzheimer’s were cleared from the brain.

The move is a setback for Alzheimer’s sufferers and their advocates, who are urging the FDA to take swift action to do everything possible to approve the drug.

Professor Robert Howard, an expert in geriatric mental health at University College London, said: “This is very bad news for Lilly.

“The longer lecanemab (competitor) has a license but donanemab does not, the more the available (but limited) US capacity to treat patients with amyloid antibodies will be blocked (for 18 months) by the first drug to come to market.”

Donanemab belongs to a growing class of drugs that aim to reduce clumps of a sticky protein called beta-amyloid, which is believed to be the main driver of this particular type of dementia.

In the company’s clinical trial, the monthly injection, 60 percent of patients showed a slowdown in cognitive decline after a year of taking it based on the criteria of one type of symptom rating scale, and by 46 percent based on another scale.

Among early Alzheimer’s patients whose brain scans showed low or medium levels of a protein called tau, the drug was found to slow clinical deterioration by 35 percent.

Tau levels are another marker of how far the disease has progressed. There is debate in the field of dementia research about whether amyloid buildup is the cause of the disease or whether it is tau.

This protein undergoes abnormal changes in the brain and becomes entangled with other protein chains. The buildup of these tangles prevents brain cells from communicating effectively with each other.

The FDA’s decision was a blow to advocates like the Alzheimer’s Association.

A spokesperson for the advocacy group said: ‘While the decision comes as a surprise at this stage of the review process, the safety and proper administration of treatments is paramount.

“On behalf of everyone who could benefit from this treatment, we strongly urge the FDA to act quickly in this next stage of its review.”

The drug, like Biogen’s similar treatment Leqembi, is intended for people with mild cognitive impairment in the early stages of Alzheimer’s. About six million Americans suffer from the disease and approximately 300,000 They are in the early stages and would likely qualify for the drug.

The field of developing Alzheimer’s treatments has been plagued by safety failures, including brain bleeds and inflammation, as well as doubts whether they actually work.

Previous studies have shown only slight declines in the rate at which the disease weakens the mind, raising doubts in the minds of reviewers tasked with approving or rejecting drug companies’ applications.

Still, there is plenty of evidence showing that some of the nearly 1,800 people enrolled in Lilly’s trial have benefited from the drug, including Connecticut native Jim Sirois, now 68.

He was diagnosed with early-onset Alzheimer’s in 2020 after having difficulty speaking and becoming so forgetful that he couldn’t remember where he had been the day before. But his wife said the drug effectively stopped the condition throughout 2023.

Sue Sirois, 64, said: “I compare Jim’s decline to that of a friend I know who was diagnosed with Alzheimer’s at age 56. She died recently and only lasted six years, while Jim has been around for more than three years. and it’s still pretty.” very similar to what he was.

Other patients in trials of the drug praised it because it helped them continue doing “normal things” like going to class and doing laundry.

Myra Garcia, 64, of Southern California, is also receiving donanemab after being enrolled in a separate trial. She told DailyMail.com that she would get the drug approved tomorrow if she could, adding that “it has worked wonderfully.”

He added: ‘I’m in three choirs, my church choir, the Hillcrest choir and a Bach choir.

“I also work out with my husband, we do something together every morning, but then I cook, do laundry and things that everyone does.”