The agency previously granted Leqembi, developed and manufactured by Eisai and Biogen, accelerated approval before evaluating late-stage clinical trial data earlier this year. The drug moderately slowed study participants’ cognitive decline compared to a placebo, according to the data, but it came with potentially serious side effects, including brain swelling and bleeding.
Under the accelerated approval, the Centers for Medicare and Medicaid Services said they will cover Leqembi only for those participating in a randomized clinical trial, in which no one was enrolling. The Medicare coverage determination was against the Veterans Health Administration, it said it would cover the drug for its beneficiaries without restriction.
About 6.7 million people in the United States have Alzheimer’s and there are few treatments for the disease. Aduhelm, a drug from Biogen that received accelerated approval in 2021, works similarly to Leqembi by targeting proteins thought to be a cause of the disease. But both drugs have received little uptake due to restrictions on Medicare coverage and concerns by patients and health care providers about evidence of its modest benefits. There are five existing treatments for Alzheimer’s that treat some of the symptoms of the disease, but do not slow the progression of the disease.
« Today’s action is the first verification that a drug targeting the underlying disease process of Alzheimer’s has shown clinical benefit, » said Teresa Buracchio, interim director of the Office of Neuroscience at the Center for Drug Evaluation and Research at the University of California. FDA. « This confirmatory study verified that it is a safe and effective treatment for patients with Alzheimer’s disease. »
Lawmakers and advocacy groups have lobbied CMS to reduce or lift coverage restrictions for Leqembi and similar drugs to ensure maximum uptake by patients. In June, CMS said it would make a national patient registry available on its website and ask doctors for only basic patient information they routinely collect, such as whether the patient is taking certain medications that could increase the risk of side effects and how a patient’s cognition progresses over time.
« With the FDA’s decision, CMS will broadly cover this drug while continuing to collect data that will help us understand how the drug works, » CMS Chiquita Administrator Brooks-LaSure said in a statement.
What’s next: Eisai estimates that about 10,000 patients will receive Leqembi by the end of March 2024.