Leqembi is the first drug approved by the FDA to treat Alzheimer's: what it is about

Photo: Bencemor / Shutterstock

The US Food and Drug Administration (FDA) made a decision that is likely to have a major impact on the Alzheimer's disease research landscape this week.

The agency issued a traditional full-fledged approval of the drug Leqembi, developed by the companies Eisai and Biogen. The drug is the first of its kind to receive such approval and is intended to slow the progression of neurodegenerative disease.

Leqembi is one of several antibody-based drugs that target amyloid beta, a protein that plays a role in Alzheimer's.

What is it about

In people with the disease, a misfolded form of amyloid builds up in the brain over time, causing the development of resistant clumps called plaques.

Scientists believe that these plaques, along with the buildup of another misfolded protein called tau, help gradually destroy the size of the brain. By breaking down or preventing the formation of plaques, it is hoped that these drugs can stop or slow the cognitive decline of people.

an effective medicine

In January 2023, the FDA issued accelerated approval for Leqembi. With these go-aheads, companies are allowed to present only indirect evidence that their drug will be clinically significant for patients, in this case, the reduction of amyloid plaque.

But companies still need to collect data and eventually confirm the clinical benefits of a drug to receive traditional approval. And it seems that Leqembi has now reached that benchmark.

"Today's action is the first verification that a drug targeting the underlying disease process of Alzheimer's disease has shown clinical benefit in this devastating disease," said Teresa Buracchio, acting director of the Center for Evaluation and Evaluation's Office of Neuroscience. FDA Drug Investigation, in a statement issued Thursday.

Photo: Getty Images

In the pivotal 18-month clinical trial that won Leqembi's approval, the drug was found to slow the progression of cognitive decline by 27% in patients compared to those taking placebo. The patients also performed better on tests of their daily functioning and had lower levels of amyloid in their brains.

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