Lilly girding for questions over Alzheimer’s treatment from FDA committee

It’s been a roller-coaster ride for three decades at Eli Lilly and Co. in trying to develop a treatment for Alzheimer’s disease.

After spending more than $3 billion on various experimental drugs over the years, only to see most of those efforts fail, the Indianapolis-based drugmaker is tying its hopes to its latest drug, a monthly injectable called donanemab.

But donanemab, which Lilly has called a potential breakthrough for slowing decline in cognition and daily function in patients with early symptoms of Alzheimer’s disease, will face a major test next month.

Analysts say the drug, if successful, could top sales of $5 billion annually.

The U.S. Food and Drug Administration is convening a panel of independent experts on June 10 to discuss donanemab’s safety and effectiveness, including the results in patients in a clinical trial.

Lilly has touted the drug for more than three years for its clinical results in slowing decline in cognition and daily function and for clearing large amounts of sticky plaque, known as beta amyloid, from the brain, as measured by imaging. The plaque, which builds up between the nerve cells of the brain, is one of the hallmarks of Alzheimer’s disease.

Now, Lilly officials are girding for the scrutiny of independent experts, who will offer a recommendation to the FDA later this year on the drug’s approval.

“We are incredibly confident in donanemab potential and the fact that it offers very meaningful benefits to people with early symptomatic Alzheimer’s disease and just the overall approvability of the package,” Anne White, president of Lilly Neuroscience, told investors in an earnings conference call on April 30.

Lilly officials said earlier this year they were not expecting the FDA to refer donanemab to its Peripheral and Central Nervous System Drugs Advisory Committee to discuss the results of a Lilly clinical trial.

The results of that trial, known as TRAILBLAZER-ALZ 2 were published in the Journal of the American Medical Association. The trial enrolled 1,736 participants across eight countries.

Lilly said the patients were selected based on cognitive assessments in conjunction with amyloid plaque imaging and tau staging by positron emission tomography imaging. The patients ranged in age from 60 to 85, with early symptomatic Alzheimer’s disease.

The FDA advisory committee’s exact questions to be discussed are not yet known, but the FDA will publish its materials for the meeting two days in advance, which will include the questions and the agency’s take on the issues, according to Fierce Pharma, an industry new site. Lilly will also provide a rebuttal.

Lilly has said the key risk associated with donanemab is amyloid related imaging abnormalities, which can be serious and life-threatening. Other reported risks included infusion-related reactions, headache and nausea.

The company had hoped to get a green light on donanemab in the first half of this year, but that is likely to be delayed until later in 2024.

“In the meantime, though, this is not time loss,” Lilly’s White said. “We’ll continue to make sure the health care system is ready. We’re going to make sure that we launch into an even stronger market with potential approval.”