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 early stages of mental illness.
Eisai and Biogen said on Saturday that the Japanese drugmaker had applied for full approval of the drug from the FDA.
The drug, which will be sold under the brand name Leqembi, belongs to a class of treatments that aim to slow the progress of the neurodegenerative disease by removing sticky clumps of the toxic amyloid beta protein from the brain.
Almost all previous experimental drugs using the same approach had failed.
“Today’s news is incredibly important,” said Dr. Howard Fillit, chief scientific officer of the Alzheimer’s Drug Discovery Foundation. “Our years of research into what is possibly the most complex human disease is paying off and gives us hope that we can make Alzheimer’s not only treatable, but preventable.”
Eisai said the drug would be launched at an annual price of $26,500. Biogen shares, which had been halted, rose 3% to $279.40.
The Japanese company said it also planned to apply for marketing authorization for Leqembi in Japan and the European Union at the end of its business year on March 31, hoping to win regulatory approval. Japanese later this year.
Eisai estimated that the number of US patients eligible for the drug would reach about 100,000 within three years, gradually increasing from there in the medium to long term.
“Our assumption is that the number of global patients eligible for the drug will grow to around 2.5 million by 2030,” Eisai Chief Executive Haruo Naito told reporters and analysts in Tokyo on Saturday.
“The new drug may not generate a significant profit immediately after launch, but it will start to contribute to our profit in the second half of the second year or the third year,” he said without giving a specific figure.
Dr. Erik Musiek, a neurologist at the University of Washington at Barnes-Jewish Hospital, said he was “pleasantly surprised” by the price of the drug.
“Given the market and the fact that we don’t have other good disease-modifying treatments, I think it’s in the realm of what I would expect,” he said.
Initial patient access will be limited by a number of factors, including reimbursement restrictions for Medicare, the US government’s insurance program for Americans age 65 and older who make up about 90% of people who may be eligible for Leqembi.
“Without the Centers for Medicare and Medicaid Services (CMS) and insurance coverage … access for those who might benefit from the newly approved treatment will be available only to those who can pay out of pocket,” he said. said the Alzheimer’s Association in a statement.
Leqembi was approved under the FDA’s expedited review process, an expedited pathway that speeds access to a drug based on its impact on biomarkers related to the underlying disease believed to predict clinical benefit.
“This treatment option is the latest therapy to target and affect the underlying Alzheimer’s disease process rather than just treating the symptoms of the disease,” said Billy Dunn, the FDA’s chief of neuroscience, in a communicated
CMS said Friday that current coverage restrictions for drugs approved under the fast track could be reconsidered based on its ongoing review of available information.
If the drug receives traditional FDA approval, CMS said it would offer broader coverage. Eisai officials have said the company plans to submit data from a recent successful clinical trial in 1,800 patients as the basis for a full standard review of Leqembi.
The CMS decision was largely in response to a previous Alzheimer’s treatment from Eisai and Biogen. Aducanumab, sold under the brand name Aduhelm, won fast-track approval in 2021 with little evidence that the drug slowed cognitive decline and despite objections from outside FDA experts.
Biogen initially cost Aduhelm $56,000 a year before cutting the price in half. 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 will have to undergo tests to show that they have amyloid deposits in the brain, either through brain imaging or a spinal tap. They will also have to undergo regular MRIs to monitor brain swelling, a potentially serious side effect associated with this type of drug.
The drug’s label says doctors should use caution if lecanemab patients receive blood thinners. That could be a safety risk, according to an autopsy analysis released this week of a lecanemab patient who had 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 early Alzheimer’s patients by 27% compared with a placebo. Almost 13% of patients treated with Leqembi in the trial had brain swelling.
Dr. Babak Tousi, a neurogeriatrician at the Cleveland Clinic, said the approval will make a “huge difference” in the field because it is based on biomarkers rather than just symptoms.
“It will change the way we diagnose Alzheimer’s disease, more accurately,” he said.
Tousi acknowledged that the drug’s benefit will likely be modest. “However, it is a benefit that we could not achieve” before this approval.
Deena Beasley in Los Angeles and Bhanvi Satija in Bangalore; Additional reporting by Jaiveer Shekhawat in Bangalore and Yuka Obayashi in Tokyo; Editing by Bill Berkrot, David Gregory, William Mallard and Tomasz Janowski
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