FDA grants accelerated approval to Alzheimer's drug for people with early stage disease
The FDA has granted accelerated approval to a new Alzheimer's treatment, called Leqembi, the first drug of its kind with clear evidence it can slow cognitive decline in people with early stages of the disease.
"Alzheimer's disease immeasurably incapacitates the lives of those who suffer from it and has devastating effects on their loved ones," said Dr. Billy Dunn, director of the Office of Neuroscience in the FDA's Center for Drug Evaluation and Research, in a statement. "This treatment option is the latest therapy to target and affect the underlying disease process of Alzheimer's, instead of only treating the symptoms of the disease."
Patients and doctors welcomed the news as a promising step forward for a disease that has proven stubbornly difficult to treat.
"This is a really exciting incremental development in the options we have to offer the millions of Americans affected by Alzheimer's disease," said Dr. Leah Croll, assistant professor of neurology at the Lewis Katz School of Medicine at Temple University.
About 1 in 9 people ages 65 and older have Alzheimer's, according to the Alzheimer's Association.
Still, the drug, called Leqembi, is far from a cure.
"It is not that you take this medication and your memory gets better," said Dr. Babak Tousi, lead investigator at the Cleveland Clinic study site and an associate professor of neurology and medicine at Lerner College of Medicine at Cleveland Clinic.
"It is a newer concept for many patients. It is not treating the symptoms but it slows down the decline," Tousi said.
And it comes with potentially significant side effects for some patients.
"There are some important safety concerns here, particularly bleeding in the brain, so patients will need to discuss those risks on a case-by-case basis with their doctors," Croll said.
A large study found that people in the early stages of Alzheimer's disease who took the drug saw a 27% slower rate of cognitive compared to people who took a placebo treatment.
This positive data, published in November, made Leqembi the first drug of its kind to produce such a positive trial result.
"This difference will likely translate to a longer period of independent living for patients," Tousi said.
Another drug, called Aduhelm, was controversially approved in 2021 based on mixed evidence of its efficacy.
Because the Centers for Medicare and Medicaid Services declined to cover Aduhelm in most cases, it has subsequently seen very low uptake. The Aduhelm approval decision was later the subject of a congressional investigation due to its questionable efficacy.
Leqembi was also approved through the FDA's conditional accelerated approval pathway, which takes into consideration the significant unmet medical need for treatments.
Accelerated approval means that the drug will be available for doctors to prescribe, but Biogen and Eisai -- the companies that make Leqembi -- will need to request full approval based on additional studies.
At this point, it's unclear whether Leqembi would be covered by most public and private insurance providers under accelerated approval, or if that would come later, after full approval.
"There's still a lot we need to learn about Leqembi, but overall, this is encouraging news. I'm hopeful that this is just the beginning, and that we will have even more options to offer these patients in the future," Croll said.
