FDA panel’s endorsement of Alzheimer’s drug paves the way for expanded usage.

A panel of health advisers in the United States has unanimously recommended full approval of the Alzheimer’s drug, Leqembi, marking a critical step toward extending insurance coverage to elderly Americans experiencing early stages of the disease.

The drug was granted conditional approval by the Food and Drug Administration (FDA) in January, following preliminary research which suggested that it could slow the progression of Alzheimer’s by several months. The FDA is now evaluating more definitive outcomes to determine whether Leqembi should receive comprehensive approval from the agency.

The panel’s verdict holds particular significance, as insurers have hesitated to cover the infused treatment until it receives full FDA approval.

In a 6-0 vote, the FDA panel of external experts confirmed that a large-scale company study established the drug’s efficacy in patients with mild or early Alzheimer’s. Although this vote is not binding, it serves as a recommendation for full approval, with the FDA scheduled to make a final decision by July 6th.

The FDA granted initial approval for Leqembi through the accelerated approval program, which permits early access to drugs based on scientific evidence indicating potential patient benefits. The drug, developed by Eisai and Biogen, has been shown to clear amyloid brain plaques associated with Alzheimer’s disease.

A panel of FDA experts examined updated data from a study involving 1,800 patients, which suggested that those taking Leqembi experienced a modest reduction in cognitive decline compared to those taking a placebo.

Dr. Merit Cudkowicz of Harvard Medical School noted that these changes were “meaningful” for patients with Alzheimer’s, which currently lacks effective treatments.

While drugs approved through the accelerated pathway may be withdrawn if their benefits are not confirmed, this is a rare occurrence. Continued research and full approval would allow Leqembi to remain on the market indefinitely.

The process of converting an accelerated approval to full FDA approval typically receives little attention and rarely involves convening advisers. However, concerns around the cost and effectiveness of plaque-targeting drugs like Leqembi have drawn new scrutiny from academics, members of Congress, and health insurers.

For instance, Medicare, a federal health program that covers 60 million seniors, has essentially blocked coverage of Leqembi and a similar drug called Aduhelm pending full FDA approval. This policy, which is unusual, was introduced last year because of concerns that Aduhelm, which is priced at $28,000 per year, would drive up healthcare costs for Medicare recipients.

The federal program provides health insurance for the vast majority of people with Alzheimer’s, and private insurers tend to follow its lead.

Leqembi is priced similarly at $26,500 per year and the handful of patients who have received it to date have mainly had to pay out of pocket.

Facing pressure from Alzheimer’s advocates and patients, Medicare’s administrator, Chiquita Brooks-LaSure, has made clear the program will immediately begin covering the drug if it gets FDA’s full OK.

But last week she announced extra requirements even after Medicare coverage begins: All patients receiving the drug will need to be enrolled in a federal registry to track Leqembi’s safety and effectiveness. That approach is occasionally used for complex new medical devices, but rarely for drugs.

Advocacy groups, including the Alzheimer’s Association, have criticized the move and lobbied the government for months to start covering Leqembi, which they believe could help thousands of Americans. The drug is the first to convincingly slow Alzheimer’s progression by targeting the underlying biology of the disease. While the delay in progression is about five months on average, some experts disagree on whether this is enough to make a meaningful difference in patients’ lives.

Despite this, the FDA’s decision to grant accelerated approval to Leqembi has been seen as a major breakthrough in the treatment of Alzheimer’s. It paves the way for other drugs aimed at combating the disease by targeting brain plaques, and underscores the importance of continuing to invest in research on Alzheimer’s and other forms of dementia.

Despite the potential side effects, most of the FDA panelists were impressed by the results of Eisai’s study, which showed significant improvements in cognitive ability and functionality, as well as reduced burden for caregivers. The 18-month study used a scale to measure key indicators of cognitive function and found that patients receiving Leqembi declined more slowly than those who received a placebo.

The drug has been associated with potentially serious side effects, such as brain swelling and small brain vessel bleeds, and three patients taking Leqembi died during the study, two of whom experienced a stroke linked to brain bleeding. However, FDA reviewers noted that it was unclear whether the drug played a role in these deaths due to other underlying factors affecting the patients, including the use of blood-thinning medications that can increase the risk of bleeding.

Panel chair Dr. Robert Alexander of the University of Arizona acknowledged the potential adverse effects of the drug but stated that they are monitorable and that the benefits of the drug are clear.

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