USA – The US Food and Drug Administration (FDA) Advisory Committee (AdCom) has unanimously voted to approve Eli Lilly’s anti-amyloid therapy donanemab for the treatment of Alzheimer’s disease. 

This decision marks a significant step forward for the drug, which has been under regulatory review since its submission in January 2023.

Donanemab is a monoclonal antibody designed to ablate amyloid plaques that aggregate in patients with Alzheimer’s disease. 

The therapy was evaluated in the Phase III TRAILBLAZER-ALZ 2 (NCT04437511) study, demonstrating that donanemab slowed disease progression in patients with high tau levels. 

This protein accumulates as neurofibrillary tangles as the disease advances. 

The study found that participants who were given donanemab declined 22% slower than those who were in the control group, as measured by a rating scale that combines two well-known scoring systems for evaluating the severity of Alzheimer’s symptoms.

The FDA is not mandated to follow the AdCom’s verdict but has historically done so. 

The agency raised questions on the pivotal study’s trial design as well as the safety and efficacy data within the enrolled population, leading to the convening of the advisory committee meeting. 

The committee concluded that donanemab’s benefits outweigh its risks, despite some concern over the drug’s safety. 

The FDA is expected to act on the committee’s recommendation in the coming months.

If approved, donanemab will join Eisai and Biogen’s marketed therapy, Leqembi (lecanemab), which shares a target for modifying disease pathology. 

The drug’s unique dosing schedule, which allows for discontinuation of treatment once amyloid levels drop below a certain threshold, was a key aspect of the committee’s discussion. 

While some panelists described this design as innovative, they still questioned how it would translate to real-world treatment and what would happen if amyloid began accumulating again in patients’ brains.

The committee also focused on a type of brain swelling and bleeding, known as ARIA, that occurred in people treated with donanemab and other drugs like it. 

ARIA is quite often asymptomatic and is only identified via magnetic resonance imaging. 

Lilly executives said that, later in the trial, they were able to better identify asymptomatic cases by having patients undergo more frequent MRIs. 

In these people, the company delayed additional drug infusions until brain swelling subsided to prevent it from becoming more serious.

Patients, caregivers, and physicians testified at the hearing, almost all of whom asked the committee to recommend an approval. 

One patient who participated in Lilly’s trial described an infusion-related side effect that ended with an emergency room visit. 

However, the patient concluded that donanemab’s benefits still counterbalanced its risks and decided to remain in the study.

The committee’s favorable recommendation removes a hurdle to Eli Lilly winning regulatory approval. 

If approved, donanemab would be the third anti-amyloid drug since 2021 to win the FDA’s blessing after Aduhelm and Leqembi. 

The drug’s approval would be a significant milestone for Lilly, which has been working on Alzheimer’s treatments for decades. The company’s shares rose 1.8 percent Monday, as panel members voted.

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