New Alzheimer's Drug Leqembi Will Be Out of Reach for Most Patients
By Joseph Walker
A sweeping Medicare rule issued last year will keep the newly approved Alzheimer's disease drug Leqembi out of reach of most U.S. patients for months to come.
The Food and Drug Administration on Friday approved Eisai Co. and Biogen Inc.'s Leqembi, known generically as lecanemab, for the treatment of people with early-stage Alzheimer's disease, the vast majority of whom are insured by Medicare. However, Medicare won't pay for the drug unless patients are enrolled in government-sanctioned clinical trials, and no such studies are ongoing or planned.
The Alzheimer's Association patient-advocacy group asked the Centers for Medicare and Medicaid Services in December to reconsider its policy, a process that could take as long as six to nine months if it chooses to do so.
As many as 85% of patients who could benefit from Leqembi are insured by Medicare, said Ivan Cheung, Eisai's global Alzheimer's disease officer. Eisai projects that 100,000 patients could be using the drug by its third year on the market, assuming that Medicare officials lift coverage restrictions, Mr. Cheung said.
CMS said Friday it "is examining available information and may reconsider its current coverage based on this review."
Biogen and Eisai priced Leqembi at $26,500 a year for the typical patient.
"It's clear there won't be coverage on day one and that's never been true of any FDA approved drug before," said Robert Egge, chief public policy officer of the Alzheimer's Association. "It's going to be very difficult if not impossible for Medicare beneficiaries to get coverage."
The holdup stems from the unusual move by Medicare officials last year to issue a rule restricting routine payment of drugs that target the protein amyloid in the brains of Alzheimer's patients.
They made the move following the approval in 2021 of Aduhelm, also made by Biogen and Eisai, based on uncertain clinical trial evidence that it was effective at slowing progression of Alzheimer's.
The agency had said its policy leaves drugmakers enough time to organize clinical trials that will answer questions the agency has about the safety and effectiveness of their drugs in Medicare patients, who tend to have more health problems than patients who enroll in industry studies.
Drugmakers and patient-advocacy groups criticized the rule for prematurely deciding the fate of similar drugs still in testing. Aduhelm was a unique scenario because Biogen officials had mistakenly ended two large clinical trials early, and were left with inconclusive results, some companies and doctors say.
Medicare officials moved too fast in judging medicines such as Leqembi based on the Aduhelm results, said Stephen Salloway, a professor of neurology and psychiatry at Brown University.
"It seemed unusual they'd make a coverage decision about future drugs when they haven't seen the results," said Dr. Salloway, who has been a paid consultant to drugmakers including Eisai and Biogen.
The FDA granted so-called accelerated approval to Leqembi based on a small, mid-stage study showing the drug significantly reduced levels of the protein amyloid in the brains of people with early Alzheimer's.
The agency says reducing amyloid is likely to predict a clinical benefit in patients, but the approval is conditioned on the benefit being proven in a follow-up study.
Eisai, which has led Leqembi's development, says it obtained the proof late last year from a large confirmatory study published in the New England Journal of Medicine showing that the drug slowed cognitive decline by 27% compared with a placebo. The company plans to file for full approval based on the study.
Eisai has been in talks with CMS officials in the lead up to the FDA approval decision, and the company plans to seek a reconsideration of the payment policy after it files for full approval, Mr. Cheung said.
If CMS changes course, Leqembi's use would be limited, Mr. Cheung said, because many Alzheimer's patients are never clinically diagnosed by a doctor, and patients with early-stage disease can be more difficult to identify than sicker patients.
"People think there are going to be a lot of people on these drugs and there's not," Mr. Cheung said.
Eli Lilly & Co. is also expecting an FDA decision on its anti-amyloid drug donanemab early this year. The company is preparing to introduce the drug in the U.S., but is expecting slow uptake unless the CMS coverage decision is liberalized, said Anne White, president of Lilly's neuroscience division.
"The use is going to be very limited in the short term," said Ms. White. "Without Medicare coverage, I think we have limited expectations."
Doctors, meanwhile, are fielding increasing calls from patients asking about Leqembi. Richard Isaacson, a preventive neurologist and researcher at the Institute for Neurodegenerative Disease of Florida, said that Leqembi requires a significant amount of effort and time on the part of patients and their family members.
Patients will need to come to a doctor's office every two weeks to get a drug infusion, as well as frequent magnetic resonance imaging, or MRI, scans in the first months of treatment to monitor for the risk of brain bleeding and swelling. Patients also need to know that they are at higher risk of such side effects if they carry a gene variant called APOE4 that is linked to Alzheimer's disease.
"This isn't just a pill you take everyday," said Dr. Isaacson.
Write to Joseph Walker at email@example.com
(END) Dow Jones Newswires
January 07, 2023 09:00 ET (14:00 GMT)
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