Alzheimer’s drug from Eisai and Biogen gets FDA accelerated approval

The first treatment clearly shown to slow the advance of brain-wasting Alzheimer’s disease has secured initial approval for commercial use from US medicines regulators.

lecanemab developed by Japanese drugmaker Eisai with US partner Biogen and targeting patients in the earliest stages of the mind-wasting disease was approved under the Food and Drug Administration’s (FDA) accelerated pathway.

Accelerated approvals are typically given to drugs for cancer and other dire diseases based on promising early results, in order to get medicines to patients sooner while more definitive confirmatory trials are conducted. In an unusual situation, Eisai completed the confirmatory trial for Leqembi before the accelerated approval, which will allow it to apply for full approval right away.

The drug, to be sold under the brand Leqembi, belongs to a class of treatments that aims to slow advance of the neurodegenerative disease by removing sticky clumps of the toxic protein beta amyloid from the brain. Nearly all previous experimental drugs using the same approach had failed.

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While Iecanemab is far from a cure, a large 18-month trial of the drug, which is given by infusion, showed it slowed the rate of cognitive decline in patients with early Alzheimer’s by 27 per cent compared to a placebo. The accelerated approval is based on data showing the drug’s ability to remove amyloid, and Eisai said it plans to submit data from a recent successful clinical trial in 1,800 patients showing the impact on cognition to get full FDA approval.

“Today’s news is incredibly important,” said Dr Howard Fillit, chief science officer of the Alzheimer’s Drug Discovery Foundation. “Our years of research into what is arguably the most complex disease humans face is paying off and it gives us hope that we can make Alzheimer’s not just treatable, but preventable.”

Eisai said the drug would launch at an annual price of $26,500 (€24,989). The company estimated that the number of US patients eligible for the drug would reach around 100,000 within three years, increasing gradually from there over the mid- to long-term.

Biogen shares rose 5 per cent on the news in New York. Eisai’s American depositary receipts rose as much as 12 per cent.

Dr Erik Musiek, a Washington University neurologist at Barnes-Jewish Hospital, said he was “pleasantly surprised” by the drug’s price. “Considering the marketplace and the fact that we have no other good disease-modifying treatments, I think it’s in the ballpark of what I would expect,” he said.

Initial patient access will be limited by a number of factors including reimbursement restrictions by Medicare, the US government insurance program for Americans aged 65 and older who represent some 90 per cent of individuals likely to be eligible for Leqembi.

Second chance

The FDA clearance could be a chance for the companies to bounce back after the controversial approval of their earlier Alzheimer’s drug, Aduhelm, in 2021. Outside medical experts advised the FDA not to allow that drug on the market because the results from two big trials conflicted on how the drug impacted brain function.

Last week, US congressional investigators said in a report that the approval process for Aduhelm was “rife with irregularities”, citing Biogen’s close collaboration with FDA staff.

Biogen initially priced Aduhelm at $56,000 per year before cutting the price in half. With limited acceptance and insurance coverage, sales were only $4.5 million in the first nine months of 2022.

In contrast, the results from lecanemab’s efficacy trial were unambiguous, though the magnitude of the benefit seen in the company’s 18-month study was modest.

“Without Centers for Medicare & Medicaid Services (CMS) and insurance coverage ... access for those who could benefit from the newly-approved treatment will only be available to those who can pay out-of-pocket,” the Alzheimer’s Association said in a statement.

The CMS decision was largely in response to the controversial approval of Aduhelm. The CMS said on Friday that current coverage restrictions for drugs approved under the accelerated pathway could be reconsidered based on its ongoing review of available information. If the drug receives traditional FDA approval, CMS said it would provide broader coverage.

Lecanemab is intended for patients with mild cognitive impairment or early Alzheimer’s dementia, a population that doctors believe represents a small segment of the estimated six million Americans currently living with the memory-robbing illness.

To receive the treatment, patients will need to undergo testing to show they have amyloid deposits in their brain – either through brain imaging or a spinal tap. They will also need to undergo periodic MRI scans to monitor for brain swelling, a potentially serious side effect associated with this type of drug. Nearly 13 per cent of patients treated with Leqembi in the trial had brain swelling.

The medicine’s label says doctors should exercise caution if lecanemab patients are given blood thinning drugs to prevent clots. This could be a safety risk, according to an autopsy analysis published this week of a lecanemab patient who had a stroke and later died.

“Alzheimer’s disease immeasurably incapacitates the lives of those who suffer from it and has devastating effects on their loved ones, said Billy Dunn, director of the office of neuroscience in the FDA’s Center for Drug Evaluation and Research. “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.”

The FDA’s action came as welcome news to the non-profit Alzheimer’s Association, chief executive Joanne Pike said in a statement. “By slowing progression of the disease when taken in the early stages of Alzheimer’s, individuals will have more time to participate in daily life and live independently,” she said. – Reuters / Bloomberg