The FDA just approved a new Alzheimer’s drug that’s set to be a blockbuster

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  • The FDA has just approved a new Alzheimer’s drug from Eisai and Biogen.

  • In a large study, the drug slowed the rate of cognitive decline in patients by 27%.

  • Alzheimer’s disease affects approximately 6.5 million Americans and there is no cure.

The U.S. Food and Drug Administration has approved a new drug for Alzheimer’s disease — a key step forward in the treatment of the disease.

Lecanemab, developed by Cambridge-based biotech company Biogen and Japanese pharmaceutical company Eisai, is the latest promising treatment for the devastating disease. The FDA has granted accelerated approval for the drug, which means companies will have to conduct additional follow-up studies.

The drug will be sold under the brand name Leqembi and costs about $26,500 a year.

“Alzheimer’s disease immeasurably impairs the lives of those who suffer from it and has a devastating impact on their loved ones,” Billy Dunn, director of the Office of Neuroscience at the FDA’s Center for Drug Evaluation and Research, said in a Friday statement. “This treatment option is the latest therapy to target and address the underlying disease process of Alzheimer’s disease, not just the symptoms of the disease.”

Ivan Cheung, CEO of Eisai’s US unit, told Insider on Thursday that the company plans to seek full FDA approval immediately. If the drug receives priority review, Leqembi could get full approval within six months.

In November, the two companies released long-awaited late-stage study results that showed the drug helped slow the rate of cognitive decline by 27% in Alzheimer’s patients over 18 months. The drug is intended for the treatment of people in the early stages of the disease.

In studies, side effects included swelling of the brain in around 13% of patients and reactions at the infusion site.

The agency’s approval comes after a long series of failures to lead to new treatments for a disease that affects about 6.5 million Americans and has no cure.

Analysts predict the drug could be a blockbuster for Biogen and Eisai.

Cowen analysts said in September 2022 that Leqembi could capture about 11% of the Alzheimer’s market three years after its launch, which would result in $4.3 billion in sales in the US alone. Globally, the drug could bring in $8 billion a year, predicts RBC Capital Markets.

Biogen shares were up 3.5% and Eisai shares were up 3.5%.

How the drug works in the treatment of Alzheimer’s disease

The science behind the drug is based on the amyloid hypothesis, a controversial theory from decades ago that posits that the buildup of beta-amyloid, or proteins also known as Aβ clumps, in the brain may be responsible for Alzheimer’s disease. These clumps build up in the brain and are thought to eventually kill brain cells, leading to disease progression.

But while Leqembi appears to have modest beneficial effects, other companies that have relied on the theory to develop Alzheimer’s drugs — including Eli Lilly and AstraZeneca, Pfizer and Roche — have faced a series of clinical trial failures.

Some biotech companies, such as Lexeo Therapeutics and Shape Therapeutics, have completely turned their backs on the amyloid beta hypothesis and are instead working to develop gene therapies to treat Alzheimer’s disease.

But Biogen and Eisai’s successful approval may convince skeptics that the amyloid-beta hypothesis still holds up.

Leqembi is delivered by IV every two weeks at the clinic. A spokesperson for Eisai told Insider that while he is conducting additional research to assess how long treatment should be taken, he believes Leqembi will likely be administered after 18 months, but it can be discontinued if the patient progresses beyond the early stages of Alzheimer’s disease.

Although the drug has been approved, questions remain about the cost

Although Leqembi has been approved by the FDA, the drug will face other key hurdles before it becomes widely available to patients.

Aduhelm, an Alzheimer’s drug also developed by Biogen and Eisai and approved by the FDA in 2021, has not received support from the Centers for Medicare and Medicaid Services, even after FDA approval. As a result, Aduhelm fell out commercially, and CMS only covers Aduhelm for patients in clinical trials.

A recent Congressional investigation also found that Biogen knew the high price of the drug would make the drug more inaccessible to patients and put a strain on the national Medicare budget.

While Leqembi has shown clearer data that it works in patients, it may face similar challenges in gaining coverage.

Leqembi will be on the market this month, but the vast majority of patients who could benefit from the drug in the US will not be able to access the treatment until CMS decides to reverse its policy on Alzheimer’s drugs that target the amyloid hypothesis -beta .

Cheung said in discussions with CMS, the agency made it clear that it needed full traditional FDA approval to consider lifting the current restrictions. He added that the company would request that CMS review its late-stage data in parallel with the FDA review to get CMS coverage as soon as possible.

“Hopefully before the end of this year we will be able to get wider access from CMS to lekanemab,” he said.

Marc Goodman, an analyst at SVB Securities, said in a December 18 note that there is “considerable nervousness with regard to CMS reimbursement, which remains an overhang.”

Still, he expects 2023 to be “a breakthrough year for Alzheimer’s.”

Goodman anticipates that Leqembi will receive full approval in the second half of the year, and CMS may update its Alzheimer’s drug policy based on the amyloid hypothesis in late 2023.

This article has been updated with the price of Leqembi.

Read the original article in Business Insider

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