By Julie Steenhuysen and Deena Beasley
(RockedBuzz via Reuters) – The U.S. Food and Drug Administration on Friday approved the Alzheimer’s drug lecanemab being developed by Eisai Co Ltd and Biogen Inc for patients in the early stages of the maddening disease.
Eisai and Biogen said on Saturday the Japanese drugmaker had applied for the drug’s full approval 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 progression of the neurodegenerative disease by removing the sticky lumps of toxic amyloid beta protein from the brain.
Nearly all previous experimental drugs using the same approach had failed.
“Today’s news is incredibly important,” said Dr. Howard Fillit, scientific director of the Alzheimer’s Drug Discovery Foundation. make Alzheimer’s not just curable, but preventable.”
Eisai said the drug will launch at an annual price of $26,500. Biogen shares, which had been locked up, rose 3% to $279.40.
The Japanese company said it plans to apply for marketing authorization for Leqembi also in Japan and the European Union by the end of its marketing year on March 31, with the hope of obtaining approval from the Japanese authorities by the end of this year.
Eisai estimated that the number of U.S. patients eligible for the drug will reach about 100,000 within three years, gradually increasing from there over the medium to long term.
“Our hypothesis is that the number of global patients eligible for the drug will grow to approximately 2.5 million by 2030,” Eisai CEO Haruo Naito told reporters and analysts on Saturday in Tokyo.
“The new drug may not generate a significant profit immediately after launch, but it will start contributing to our profit in the second half of the second or third year,” he said without providing any concrete figures.
Dr. Erik Musiek, a Washington University neurologist at Barnes-Jewish Hospital, said he was “pleasantly surprised” by the drug’s price.
“Considering the market and the fact that we don’t have any other good disease-modifying treatments, I think it lives up to 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’s insurance program for Americans age 65 and older which account for approximately 90% of people who might be eligible for Leqembi.
“Without the 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,” said the Alzheimer’s Association in a statement.
Leqembi was approved under the FDA’s accelerated review process, a fast-track pathway that accelerates access to a drug based on its impact on underlying disease-related biomarkers that are thought to predict clinical benefit.
“This treatment option is the latest therapy to target and influence the underlying disease process of Alzheimer’s instead of just treating the symptoms of the disease,” said Billy Dunn, FDA neuroscience officer.
CMS said Friday that current coverage restrictions for drugs approved under the fast track could be reconsidered based on the ongoing review of available information.
If the drug receives traditional FDA approval, CMS said it would provide broader coverage. Eisai officials said the company plans to submit data from a recent successful clinical trial involving 1,800 patients as the basis for a comprehensive standard review of Leqembi.
CMS’s decision was largely in response to previous Alzheimer’s treatment from Eisai and Biogen. Aducanumab, sold under the brand name Aduhelm, gained accelerated approval in 2021 with little evidence that the drug has slowed cognitive decline and despite objections from outside FDA experts.
Biogen initially valued Aduhelm at $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 doctors believe represents a small segment of the approximately 6 million Americans currently living with the memory-stealing disease.
To receive the treatment, patients will need to undergo tests to prove they have amyloid deposits in their brain, either through brain imaging or a lumbar puncture. They will also need to have periodic MRI scans to monitor for brain swelling, a potentially serious side effect associated with this type of drug.
The medicine’s label says that doctors should be careful if lecanemab patients are given blood clot prevention drugs. 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 study of lecanemab, given by infusion, the drug slowed the rate of cognitive decline by 27% in patients with early Alzheimer’s disease compared to a placebo. Almost 13% of patients treated with Leqembi in the study had brain edema.
Cleveland Clinic neuro-geriatrician Dr Babak Tousi said the approval would make a “big difference” in the field because it was 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 is likely to be modest. “However, it’s a lead we haven’t been able to get” prior to this approval.
(Reporting by Deena Beasley in Los Angeles and Bhanvi Satija in Bengaluru, additional reporting by Jaiveer Shekhawat in Bengaluru and Yuka Obayashi in Tokyo; editing by Bill Berkrot, David Gregorio, William Mallard and Tomasz Janowski)