Eisai, Biogen receives US FDA approval for Alzheimer’s drug, seeks full approval

Jan 7 (Reuters) – The U.S. Food and Drug Administration on Friday approved the Alzheimer’s drug lecanemab developed by Eisai Co Ltd ( 4523.T ) and Biogen Inc ( BIIB.O ) for patients in the earliest stages of the disease to lose the brain.

Eisai and Biogen said on Saturday that the Japanese drugmaker had applied for full FDA approval of the drug.

The drug, to be sold under the brand name Leqembi, belongs to a class of treatments aimed at slowing the progression of the neurodegenerative disease by removing sticky clumps of the toxic protein beta-amyloid from the brain.

Almost all previous experimental drugs that used the same approach had failed.

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

Eisai said the drug would launch at an annual cost of $26,500. Biogen shares, which had been halted, rose 3% to $279.40.

The Japanese company said it also plans to apply for marketing authorization for Leqembi in Japan and the European Union by the end of the business year on March 31.

Eisai estimated the number of US patients eligible for the drug will reach about 100,000 within three years, and gradually increase from there in the medium to long term.

Dr. Erik Musiek, a neurologist at Washington University at Barnes-Jewish Hospital, said he was “pleasantly surprised” by the drug’s price.

“Given the marketplace and the fact that we don’t have any other good disease-modifying treatments, I think it’s at the low end of what I would expect,” he said.

Initial patient access will be limited by a number of factors, including reimbursement limitations from Medicare, the US government’s insurance program for Americans age 65 and older who represent about 90% of individuals likely to be eligible for Leqembi.

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

Leqembi was approved under the FDA’s accelerated review process, an expedited pathway that allows faster access to a drug based on its impact on underlying disease-related biomarkers thought to predict a clinical benefit.

“This treatment option is the latest therapy to target and affect the underlying disease process of Alzheimer’s rather than just treating the symptoms of the disease,” FDA neuroscientist Billy Dunn said in a statement.

CMS said on Friday that current coverage limitations for drugs approved under the accelerated track may be reevaluated based on the ongoing review of available information.

If the drug receives traditional FDA approval, CMS said it would provide broader coverage. Eisai officials have said the company plans to submit data from a recent successful clinical trial involving 1,800 patients as the basis for a full standard review of Leqembi.

The CMS decision was largely a response to an earlier Alzheimer’s treatment from Eisai and Biogen. Aducanumab, sold under the brand name Aduhelm, won accelerated approval in 2021 with little evidence that the drug slowed cognitive decline and despite objections from the FDA’s outside experts.

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

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 6 million Americans currently living with the memory-robbing disease.

To receive the treatment, patients must undergo tests to show that they have amyloid deposits in the brain – either through brain imaging or a spinal tap. They will also need to undergo periodic MRI scans to monitor for swelling in the brain, a potentially serious side effect associated with this type of drug.

The drug’s label states that doctors should use caution if lecanemab patients receive blood clot prevention agents. This could be a safety risk, according to an autopsy analysis published this week of a lecanemab patient who suffered a stroke and later died.

In the large trial of lecanemab, which is given by infusion, the drug slowed the rate of cognitive decline in patients with early Alzheimer’s by 27% compared to placebo. Almost 13% of patients treated with Leqembi in the study had brain swelling.

Dr. Babak Tousi, a neuro-geriatrician at the Cleveland Clinic, said the approval will make a “big difference” in the field because it is based on biomarkers rather than just symptoms.

“It’s going to change how we diagnose Alzheimer’s disease, with more accuracy,” he said.

Tousi acknowledged that the benefit of the drug is likely to be modest. “Still, it’s an advantage we weren’t able to achieve” before this approval.

Reporting by Deena Beasley in Los Angeles and Bhanvi Satija in Bengaluru, additional reporting by Jaiveer Shekhawat; Editing by Bill Berkrot, David Gregorio and William Mallard

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