Amyloid, a toxic protein that slowly builds up in the brains of Alzheimer's patients
Eisai Co.'s Alzheimer's drug Leqembi gained full approval from US regulators, a move that will help widen insurance access to the first drug cleared based on its ability to slow progression of the mind-robbing disease.
The broader clearance from the Food and Drug Administration marks a milestone for treatment of the dementia that afflicts some 6 million Americans. It's expected to lead to far greater coverage by insurers and the federal Medicare program for the medicine from Eisai and its partner Biogen Inc., which received expedited approval in January. The drug costs $26,500 for annual treatment.
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Patients taking it need MRI scans to monitor for early signs of side effects that can include swelling and bleeding in the brain. The label includes a prominent black-box warning about the potential for these conditions, which are usually asymptomatic but in rare cases can lead to life-threatening complications. The warning also notes patients with a certain gene mutation called APOE4 have a higher risk of such side effects.
The drug is among the most potent of a new generation of antibodies that help clear amyloid, a toxic protein that slowly builds up in the brains of Alzheimer's patients. Last fall, Leqembi became the first drug to clearly slow the disease in a large trial that showed patients who received it declined at a 27% slower rate over 18 months compared to a sham treatment.
Related: Health insurers balk at Biogen's $56,000-a-year Alzheimer's treatment
Researchers have debated for decades over the degree to which amyloid contributes to Alzheimer's, and numerous drugs that fight the abnormal deposits failed in trials or showed ambiguous results. While far from a cure, Leqembi showed that removing amyloid could have a measurable effect on disease progression over long periods of time.
The Alzheimer's Association, which pushed heavily for approval and coverage for amyloid-lowering drug, called Leqembi "the first Alzheimer's treatment granted traditional approval that changes the underlying course of the disease." It will help people at early stages of the disease maintain their independence for longer, said Joanne Pike, the advocacy group's chief executive officer.
The drug's new label "looks relatively benign and overall favorable, with no major surprises," RBC Capital Markets analyst Brian Abrahams said in a note to clients.
Scant use
The drug received accelerated US approval in January. But because Medicare earlier ruled that it wouldn't cover amyloid-lowering drugs without full approval based on documented clinical benefit, few patients have received Leqembi so far.
In June, the Centers for Medicare & Medicaid Services indicated that broader coverage for Leqembi would begin immediately upon full approval, although doctors will have to enroll patients getting the drug in a registry to track treatment details and collect additional evidence on the drug's risks and benefits.
The same month, the drug sailed through a meeting of outside advisers to the FDA who unanimously recommended full approval. Still, some members said they worried about potential risks among certain patients, including those taking anticoagulants.
In a statement, CMS confirmed it would now cover the drug. "With FDA's decision, CMS will cover this medication broadly while continuing to gather data that will help us understand how the drug works," CMS Administrator Chiquita Brooks-LaSure said.
The agency Thursday on its website cited research that would meet its registry requirement, an observational study to track patients' outcomes over two years. Clinicians will be required to submit data every six months.
"Today's action is the first verification that a drug targeting the underlying disease process of Alzheimer's disease has shown clinical benefit in this devastating disease," Teresa Buracchio, acting director of the Office of Neuroscience in the FDA's Center for Drug Evaluation and Research, said in a statement.
Leqembi is not simple to use: It's infused every two weeks by a medical provider. In addition to regular MRI scans, patients need to get a separate test before treatment to confirm they have amyloid in the brain. The drug has only been shown to help patients at the earliest stages of the disease.
It could soon have competition. In May, Eli Lilly & Co. said its similar antibody drug, donanemab, slowed the disease in a final-stage trial. It has said it planned to complete a filing for full approval by the end of the second quarter. The drug is under review for approval in both Europe and Japan.
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