The first Alzheimer’s drug proven to moderately slow the course of the disease gained full approval from the Food and Drug Administration (FDA) on Thursday.
The FDA said the approval was based on clinical trial data that showed Leqembi slows cognitive decline by about 27 percent compared to a placebo among Alzheimer’s patients who are in the early stages of the disease.
“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 FDA’s Office of Neuroscience.
The agency’s decision also triggers a change in how the Medicare program will cover the twice-monthly infusion, which costs $26,500 annually.
The Centers for Medicare and Medicaid Services (CMS) limits coverage to people who are participating in clinical trials, but full FDA approval means Medicare will broaden coverage for potentially millions more, even as Leqembi’s benefits and risks remain a source of debate.
About 6.7 million people in the United States are diagnosed with Alzheimer’s, and that number is expected to rise as the population ages. Drugmaker Eisai has said it expects about only 100,000 patients will be eligible and using the drug in its first three years on the market.
As part of its coverage decision, Medicare will require providers to collect data for patient registries about how the drug performs in real-world settings. The registry is not live, so it’s not clear yet how it will perform, but CMS has indicated it’s trying to make registry participation as easy as possible for clinicians and patients.
Clinicians will be able to submit this information through a nationwide, CMS-facilitated portal. The registry will collect information about patients such as clinical diagnosis, results from amyloid tests, and evidence of adverse events — such as brain swelling or hemorrhage, among other data.
The decision to require a registry angered industry and patient advocacy groups, who have been waging an intense lobbying campaign for broad coverage. Bipartisan lawmakers have also called for unrestricted coverage.
House Energy and Commerce Chairwoman Cathy McMorris Rodgers (R-Wash.) on Wednesday said in a statement CMS should already be covering key Alzheimer’s treatments that have been cleared under the FDA’s accelerated approval process, and if the agency grants traditional approval, “There will be no excuses for CMS to effectively deny coverage to Americans in need.”
FDA granted Leqembi accelerated approval in January, based on evidence from clinical trials that it was successful at removing brain plaque known as amyloid, a signature characteristic of Alzheimer’s disease.
The drug was approved only for people with early forms of Alzheimer’s disease, those with mild cognitive impairment or mild dementia who have been confirmed to have amyloid plaques in their brains. Identifying those patients will be a challenge, but it could change the way Alzheimer’s is treated.
“I think a whole new type of patient may end up coming to us much earlier than we tend to see,” said Jeffrey Burns, a University of Kansas Medical Center neurologist. “We’ve always stressed early diagnosis, but now we have a drug that looks to be most helpful in the earliest stages. And I think that’s going to drive home … early diagnosis, and see a lot more patients in that stage.”
Full approvals require more clinical evidence than accelerated approvals. And positive outcomes of Leqembi are tempered by a serious type of brain swelling or bleeding that afflicted 13 percent of people during the trials. Three people died.
During an advisory committee meeting last month, FDA staff said the risks do not “appear to preclude traditional approval,” but they should be considered ahead of any treatment.
The drug is not a cure and doesn’t stop people with Alzheimer’s from getting worse. Critics have said there was no real clinically meaningful impact of Leqembi, and the differences between people on the placebo and people taking the drug were minimal.
Supporters argue any delay in the progression of the disease matters because the differences in early-stage patients can amount to several additional months.
Dennis Selkoe, co-director of the Center for Neurologic Diseases at Brigham and Women’s Hospital in Boston who has consulted for Eisai, said he thinks Alzheimer’s patients and families will want to take the risk.
“While it’s not a cure by any means, it’s a robust slowing of cognitive decline. And most importantly, activities of daily living — being able to, you know, get dressed, make a cup of coffee, do a simple financial task, send a check out — are the things that matter the most to my patients,” Selkoe said.