- A new Alzheimer’s drug, Leqembi, has received approval on July 7 from the Food and Drug Administration.
- The FDA approval comes after the medication showed promise in slowing cognitive decline in a phase 3 clinical trial.
- There are reports that three people died while taking the drug during the trial. The drug may also cause brain swelling and bleeding.
- Though Medicaid will cover a substantial portion of the cost of Leqembi, some experts have said Leqembi may reduce Alzheimer’s symptoms but not improve brain function.
The drug, lecanemab, will be sold under the brand name Leqembi.
“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, the director of the Office of Neuroscience in the FDA’s Center for Drug Evaluation and Research, in a press 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.”
Some experts have questioned its effectiveness, but the medication showed promise in a phase 3 clinical trial.
Researchers said lecanemab slowed cognitive and functional decline by 27% when given to people with Alzheimer’s in the clinical trial.
However, the journal Science reported in late December that three people died while taking the drug during the clinical trial. The journal reports that the third death was a 79-year-old Florida woman who died in mid-September after developing brain swelling and bleeding.
Here’s everything you need to know about Leqembi as a potential treatment for Alzheimer’s disease.
Leqembi is the brand name of lecanemab, which is used to treat early Alzheimer’s disease. In earlier clinical trials, it was shown to lower levels of beta-amyloid plaque, a biomarker of the disease found in the brain.
“Lecanemab… is a monoclonal antibody infusion therapy that targets components of beta-amyloid, which build up… as part of the plaques and tangles that are characteristic of Alzheimer’s disease. And these new therapies effectively clear those amyloid plaques. It’s an exciting new chapter in the treatment of Alzheimer’s disease,” said Dr. Scott A. Kaiser, a geriatrician and the director of geriatric cognitive health for the Pacific Neuroscience Institute at Providence Saint John’s Health Center in Santa Monica, CA.
“We know that it clears the beta-amyloid plaque,” Kaiser told Healthline in September. “The question is whether or not that actually helps with brain function. But the idea is that these plaques are interfering with the effective communication and overall interaction between brain cells and that clearing them could have positive effects.”
The drug is given via an infusion. Side effects of the drug may include:
- infusion-related reactions
- amyloid-related imaging abnormalities (ARIA)
The drug will carry a boxed warning for patients about the potential of ARIA.
ARIA can present as swelling and potential bleeding in the brain; usually, the issue resolves over time. However, according to the FDA, it can result in brain edema, leading to seizures and other life threatening complications.
Lecanemab was granted breakthrough therapy designation by the FDA in June 2021.
This status is designed to speed up the development of new drugs that will address medical needs that are currently unmet for serious or life-threatening conditions.
However, some scientists have expressed concern that the earlier phase 2 trials of lecanemab had flaws and that the actual benefit of the drug to people could be limited.
“The phase 2B lecanemab studies were fatally flawed because the high dose versus placebo analysis (that supposedly showed some clinical benefit) was profoundly compromised,” Dr. Michael Greicius, a professor of neurology and neurological sciences at Stanford University in California, told Healthline in an earlier interview.
Greicius argued that in the phase 2B trial, people who were carriers of APOE4, a type of gene associated with an increased risk of Alzheimer’s disease, were prevented midway through the trial from receiving a high dose of the treatment.
“This means that there were many more APOE4 carriers in the placebo group (71%) than in the high dose group (30%),” Greicius explained. “This difference in percentage of APOE4 carriers is as likely (or in my view more likely) than the drug to account for the difference in clinical outcomes.”
The recent clinical trial was conducted at 235 sites in North America, Asia, and Europe between March 2019 and March 2021.
The study involved nearly 1,800 adults ages 50 to 90. All the participants had some form of early dementia or Alzheimer’s disease. Half of the participants were given lecanemab and the other half were given a placebo.
Researchers reported there wasn’t a significant difference between lecanemab and the placebo at 12 months, but at 18 months it appeared the people taking lecanemab had some clearance of amyloid and less cognitive decline.
However, researchers said participants taking lecanemab had a higher percentage of adverse events than people taking the placebo at both 12 months and 18 months.
The Centers for Medicare and Medicaid Services announced they will provide significant coverage for Leqembi.
“CMS today affirms our commitment to help people with Alzheimer’s disease have timely access to innovative treatments that may lead to improved care and better outcomes,” CMS Administrator Chiquita Brooks-LaSure said in a statment.
“With FDA’s decision, CMS will cover this medication broadly while continuing to gather data that will help us understand how the drug works. This is welcome news for the millions of people in this country and their families who are affected by this debilitating disease.”
The CMS said that people with original Medicare plan will pay 20% coinsurance of the Medicare-approved amount after meeting their Part B deductible.
The FDA’s approval of Leqembi was praised by officials at several Alzheimer’s organizations.
“This treatment, while not a cure, gives people in the early stages of Alzheimer’s disease more time to maintain their independence and do the things they love,” Joanne Pike, DrPH, Alzheimer’s Association president and CEO said in a statement.
“While we continue efforts to discover new targets and test new treatments, people living with this fatal disease deserve the opportunity to discuss and make the choice with their doctor if an FDA-approved treatment is right for them.”
The reaction was equally positive from the Alzheimer’s Drug Discovery Foundation (ADDF).
“This is encouraging news, and more importantly, the approval of Leqembi will serve as a catalyst for driving further developments and investments in the Alzheimer’s pipeline,” Howard Fillit, MD, Co-Founder and Chief Science Officer of ADDF said in a statement.
“We finally have clarity around amyloid’s modest effect on cognitive decline. Now, it is more important than ever to double down and widen our focus to developing the next generation of drugs based on the biology of aging that can lead to a combination therapy and precision medicine approach.”
A similar drug, Aduhelm, has also been cleared for use.
In 2021, Aduhelm received FDA approval as the first new treatment for Alzheimer’s disease since 2003. It received approval on the basis of the drug being effective at reducing beta-amyloid plaque.
“This approval was met with a great deal of criticism by the scientific community because there are no compelling data to show that reducing amyloid plaque is associated with improved clinical outcomes,” Greicius said.
“Lecanemab also has a similar profile of dangerous side effects related to brain swelling and brain bleeding that we see with Aduhelm, though lecanemab is probably a bit friendlier than Aduhelm on this front in that ‘only’ 10 percent of patients in the high dose groups showed these side effects [in the phase 2 trial],” Greicius added.
It’s estimated that nearly
Alzheimer’s disease is a form of dementia that can progress from mild memory loss in the early stages to the potential for a person with the disease to have difficulty engaging in conversation or responding appropriately to what is around them.
There is currently no cure for Alzheimer’s disease, and treatment options are limited.
“There aren’t a lot of alternatives, particularly when it comes to drugs. There are drugs that can boost certain levels of neurotransmitters and, otherwise, potentially enhance cognition. But they don’t modify the actual underlying disease pathology or disease course,” Kaiser said.
“There are some minor symptomatic treatments. It’s akin to cough syrup for somebody who has a cold. It doesn’t actually cure or treat the underlying cold, it can just provide some symptomatic relief. And in terms of pharmacotherapy for Alzheimer’s disease… that’s all there is. That’s all that’s been approved in decades,” he added.