Eisai and Biogen’s Leqembi received the coveted standard approval nod from the US Food and Drug Administration, making it the first Alzheimer’s treatment to do so, paving the way for the drug to be covered by more insurance companies.
First Alzheimer’s treatment
The FDA decision is a significant step forward for a fatal disease that has eluded drugmakers for decades. According to trial results, the treatment slows the progression of the brain-wasting disease by 27% in patients in the early stages of Alzheimer’s.
On Leqembi’s label, the FDA also included its strongest “boxed” safety warning, highlighting the risk of potentially dangerous brain swelling for Alzheimer’s drugs in the same class. Eisai shares fell in Tokyo trade on Friday, citing the safety warning as a negative surprise.
Leqembi is an antibody that is designed to remove sticky deposits of a protein called amyloid beta from Alzheimer’s patients’ brains.
“Today we believe is a triumph for the Alzheimer’s disease community, after so many years of hard work by so many scientists, physicians and clinical trial participants and their care partners,” Eisai’s U.S. chief executive, Ivan Cheung, said
Leqembi received “accelerated” FDA approval in January based on its amyloid-clearing ability, but coverage under the US government’s Medicare health plan for people 65 and older was limited to patients in clinical trials.
Leqembi will now be covered under standard approval, though the Centers for Medicare and Medicaid Services (CMS) is linking reimbursement to patient participation in a health agency database known as a registry. Because Alzheimer’s is a disease of aging, Medicare covers the majority of patients in the United States.
“With the 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 stated in a statement. Leqembi, which is administered intravenously, has a list price of $26,500 in the United States per year.
Dr. Babak Tousi, the Cleveland Clinic’s neuro-geriatrician primary investigator of the Leqembi clinical trials, said he expects a lot of interest in the treatment but estimates that only about 1 in 10 patients will qualify for the drug.
Tousi stated that Leqembi “is not a cure,” and that it will only slow the progression of the disease. “This is just one piece of the puzzle,” he explained.
BRAND NEW LABEL
The new label for Leqembi emphasizes the importance of monitoring patients for potentially dangerous brain swelling and bleeding caused by amyloid-lowering antibodies.
It states that the risk is higher in patients who have two copies of an Alzheimer’s gene, APOE4, and that while genetic testing is highly recommended, it is not required.
The new label for the drug includes information indicating that the use of certain anticoagulants with Leqembi has been linked to an increased risk of brain hemorrhage.
“The boxed warning seems appropriate, as the risks need to be carefully considered and discussed with patients,” said Dr. Erik Musiek, a Washington University neurologist at Barnes-Jewish Hospital, in an email. Eisai’s stock fell 5% in Tokyo trading, despite having gained roughly 50% in the previous year.
According to Jefferies analysts, the FDA’s unexpected decision to place the warning on the label could contribute to the drug’s slow sales growth, as the recommendation for genetic testing would increase costs for prospective patients.
However, Eisai CEO Haruo Naito told reporters in Tokyo that he believed that in the not-too-distant future, U.S. health insurance would likely cover the entire treatment process, including genetic testing. He also stated that the drug would be approved in Japan by the end of September.
According to a press release issued in May, the safety warning will also apply to Eli Lilly and Co’s (LLY.N) donanemab, an experimental Alzheimer’s drug that was shown to slow cognitive decline by 35% in a late-stage trial. The full findings of that study are expected later this month.
Cheung stated that Eisai is stepping up efforts to get health centers ready to use Leqembi, but she declined to say how many patients have been treated with the drug thus far.
“People living with this fatal disease deserve the opportunity to discuss and choose, with their doctor and family, whether an FDA-approved treatment is right for them,” said Joanne Pike, president and CEO of the Alzheimer’s Association, in a statement.
After an agency staff report concluded that the drug offered a meaningful benefit to patients and that safety concerns could likely be managed, an FDA committee of external advisers recently recommended traditional approval of the drug. According to the Alzheimer’s Association, more than 6 million Americans have Alzheimer’s disease.
Eisai estimates that amyloid-lowering drugs will be used in 100,000 patients in the United States during Leqembi’s first three years on the market.
Aduhelm, the first FDA-approved disease-modifying Alzheimer’s drug, was also developed by Eisai and Biogen partners, but Medicare coverage restrictions severely limited its use.
Eisai is seeking approval for Leqembi in the EU, China, Canada, the United Kingdom, and South Korea, in addition to the United States and Japan. The application has been designated as a priority review in China, as it has been in Japan.
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