FDA Approves Eisai, Biogen’s Alzheimer’s Drug Leqembi

The Food and Drug Administration on Thursday fully approved Alzheimer’s drug Leqembi, a crucial decision that will expand access to the expensive drug for older Americans.

Medicare announced shortly after FDA approval that it would now cover antibody treatment for patients enrolled in the seniors’ insurance program, although several conditions apply.

Leqembi is the first Alzheimer’s antibody treatment to receive full FDA approval. It’s also the first drug of its kind to have comprehensive coverage under Medicare.

Leqembi is not a remedy. The treatment slowed cognitive decline due to early Alzheimer’s disease by 27% over 18 months during Eisai’s clinical trial. The antibody, given twice a month by intravenous infusion, targets a protein called amyloid, which is linked to Alzheimer’s disease.

Medicare coverage is a critical step in helping older Americans with early Alzheimer’s pay for treatment. With a median income of about $30,000, most Medicare beneficiaries can’t afford Eisai’s $26,500 annual price for Leqembi without insurance coverage.

Medicare previously only agreed to cover Leqembi for patients enrolled in clinical trials after the treatment received accelerated approval in January. This policy had severely restricted access to the drug.

To be eligible for coverage, patients must be enrolled in Medicare, have been diagnosed with mild cognitive impairment or mild Alzheimer’s disease, and have a physician who participates in a data collection system the federal government has put in place to benefit and monitor risks of treatment.

Joanna Pike, president of the Alzheimer’s Association, the lobby group that advocates for people with the disease, said while Leqembi is not a cure, it will help patients in the early stages of the disease maintain their independence and go about their daily lives and spend more time with their families.

“This gives people more months to recognize their spouse, children and grandchildren,” Pike said in a statement Thursday. “It also means you have more time to drive safely, carefully manage your family’s finances and fully devote yourself to your hobbies and interests.”

However, treatment carries serious risks of brain swelling and bleeding. Three patients who participated in Eisai’s study died. FDA scientists said it was unclear whether Leqembi played a role in those deaths.

According to the FDA, Alzheimer’s disease is the leading cause of dementia in older adults and the sixth leading cause of death in the United States.

dr David Knopman, an Alzheimer’s neurologist at the Mayo Clinic in Minnesota, said Leqembi clearly showed patient benefit in the Eisai study, but cautioned that the treatment’s effectiveness was modest.

Knopman said appropriately diagnosed and informed patients should be able to make their own decision about taking Leqembi after weighing the benefits and risks of the treatment and the potential logistical challenges of finding a location for the twice-monthly infusions .

Medicare coverage

To get coverage, Medicare requires patients to find a healthcare provider that participates in a registry system that collects real-world data about the drug’s benefits and risks. The system is controversial. The Alzheimer’s Association and some members of Congress fear this requirement will create barriers to treatment.

There are concerns that the number of healthcare providers participating in such registries will be limited and that people in rural towns and other underserved communities will have to travel hours to find one.

The Centers for Medicare and Medicaid Services have established a nationwide portal to make it easier for healthcare providers to submit the required data on patients receiving Leqembi. The portal, which can be used free of charge, went online moments after the FDA’s decision on Thursday.

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Rep. Anna Eshoo of California, the senior Democrat on the House Health Subcommittee, and Rep. Nanette Barragan, D-California, in a letter to CMS last month raised concerns that patients might have trouble finding a doctor participating in the system find.

Alzheimer’s is usually diagnosed using a PET scan to detect the amyloid protein associated with the disease, or in some cases, a spinal tap. Medicare currently only covers one PET scan per life for dementia. It’s unclear if the program plans to change this policy.

There is also a concern that if Leqembi is widely accepted as a treatment and patient demand for the antibody is high, there may be too few specialists and sites to administer the infusions.

Some studies have estimated waiting times for antibody treatments like Leqembi could range from months to even years over the next decade, depending on demand.

Tomas Philipson, who advised the FDA commissioner and CMS administrator during the second Bush administration, said the registry is an unnecessary hurdle and Medicare should scrap it, but doesn’t think the requirement creates an insurmountable hurdle for patients to access Leqembi will represent.

When demand for Leqembi is high, doctors would have an incentive to participate in the registry and drug companies would want to help, said Philipson, a health economics expert at the University of Chicago.

How high the demand for Leqembi will be is uncertain, he said. Families concerned about the serious side effects may choose not to take the treatment, while others will decide the benefits outweigh the risks, he said.

High costs

The price of Leqembi and the benefit-risk profile of the treatment are controversial.

According to CMS, Medicare patients treated with Leqembi pay 20% of their medical bill after meeting their Part B deductible. According to the agency, costs can vary depending on whether the patient has additional Medicare coverage or other secondary insurance.

According to a study published in the journal JAMA Internal Medicine, patients could expect up to $6,600 in annual deductibles for Leqembi despite Medicare coverage. The treatment could cost Medicare as much as $5 billion a year depending on how many people receive the IVs, the study estimates.

Senator Bernie Sanders, I-Vt., chair of the Senate Health Committee, has called Leqembi’s price “outrageous” and wrote in a letter last month asking Health and Human Services Secretary Xavier Becerra to take action to cut costs.

Sanders said patients’ own expenses for Leqembi accounted for one-sixth of many seniors’ total annual income, noting that the high cost of the treatment could increase premiums for all Medicare beneficiaries.

According to Eisai, Leqembi’s annual list price of $26,500 is below the company’s estimate of $37,600 for the total value of treatment for each patient. The Institute for Clinical and Economic Review, a nonprofit that analyzes health care costs, estimated in April that the price could range from $8,900 to $21,500 a year.

Although Leqembi could prove costly for Medicare, Philipson said delaying treatment coverage would result in significantly higher healthcare spending as people with mild Alzheimer’s disease, which can be treated at home, progress to a more serious condition that can be treated requires expensive nursing home care.

Philipson and his colleagues at the University of Chicago estimated that a one-year delay in Medicare coverage of Alzheimer’s antibody treatments would result in $6.8 billion in increased spending. By 2040, healthcare spending would increase by $248 billion.

Clinical Benefit

Full FDA approval came Thursday after a panel of six outside advisors unanimously voted in June for the drug’s clinical benefit to patients. The board was unusually small, with some members retiring due to conflicts of interest.

The American Academy of Neurology said in a February letter to CMS that there was consensus among its experts that Eisai’s clinical trial of Leqembi was well designed and the results were “clinically and statistically significant.”

Some nonprofit groups, such as Public Citizen, a consumer advocacy organization, strongly opposed the FDA’s approval of Leqembi. A Public Citizen representative told the advisory panel that the evidence supporting the drug’s benefits does not outweigh the significant risks of brain swelling and bleeding.

And officials from the National Center for Health Research and Doctors for America, also nonprofits, told the panel that Eisai’s clinical trial did not include enough black patients, who were at higher risk of developing Alzheimer’s disease.

Leqembi has technically been approved for the US market since January, when the FDA approved the treatment under an accelerated approval process. The FDA uses expedited approvals to save time and get medicines to patients with serious illnesses faster.

But Medicare refused to pay for Leqembi at the time, demanding more evidence that the expensive treatment had real clinical benefits for patients that outweighed the risks.

The program’s cautious coverage policy follows the FDA’s controversial 2021 approval of another Alzheimer’s antibody treatment called Aduhelm, also made by Eisai and Biogen.

The FDA Advisory Committee denied approval of Aduhelm because the data did not demonstrate clinical benefit for patients. Three advisers resigned after the agency decided to approve the treatment anyway.

Knopman is one of the consultants who resigned following the FDA’s decision on Aduhelm. He said the dates for Leqembi are different. Eisai conducted a clean study that showed the antibody had modest clinical benefit for patients, Knopman said.

A congressional investigation subsequently found that Aduhelm’s FDA approval was “riddled with irregularities.”

Sanders said in his letter to Becerra that the FDA “has a special responsibility to restore public confidence after forming an inappropriate relationship with Biogen during the agency’s review of a previous Alzheimer’s drug, Aduhelm.”