FDA just approved a new blockbuster Alzheimer’s drug


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

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

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

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

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

The drug will be sold under the brand name Leqembi and carries a price tag of about $26,500 per year.

“Alzheimer’s disease is immeasurably debilitating to the lives of those who suffer from it and has devastating effects on their loved ones,” Billy Dunn, director of the Office of Neuroscience at the FDA’s Center for Drug Evaluation and Research, said Friday. Said. “This treatment option is the latest therapy to target and influence Alzheimer’s underlying disease process, rather than merely treating the symptoms of the disease.”

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

In November, the two companies published long-awaited advanced study results showing that the drug helped slow the rate of cognitive decline in Alzheimer’s patients by 27% over 18 months. The drug is aimed at treating people in the early stages of the disease.

Adverse events in the trials included brain swelling and infusion site reactions in approximately 13% of patients.

The agency’s approval comes after a long series of failures to introduce new treatments for the incurable disease that affects nearly 6.5 million Americans.

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

Analysts from Cowen said in a September 2022 note that Leqembi could take over roughly 11% of the Alzheimer’s disease market three years after launch, adding $4.3 billion in US sales alone. RBC Capital Markets estimated that globally the drug could bring in $8 billion a year.

Biogen’s shares rose 3.5% and Eisai’s shares rose 3.5%.

How does the drug work to treat Alzheimer’s?

The science behind the drug is based on the amyloid hypothesis, a decades-old controversial theory that suggests that proteins in the brain, also called beta-amyloid or Aβ clusters, may be responsible for Alzheimer’s disease. These clumps are thought to accumulate in the brain and eventually kill brain cells, leading to disease progression.

But while Leqembi appears to have modest beneficial effects, other companies that have relied on this theory to develop drugs for Alzheimer’s disease — 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 abandoned the amyloid-beta hypothesis altogether and are instead trying to develop gene therapies to treat Alzheimer’s.

But Biogen and Eisai’s successful confirmation may convince skeptics that the amyloid-beta hypothesis is still valid.

Leqembi is given through an IV every other week in a clinic. A spokesperson for Eisai’s told Insider that he believes Leqembi will likely be administered after 18 months, while conducting additional studies to assess how long treatment should last, but may be discontinued if the patient has passed the early stages of Alzheimer’s.

While the drug has been approved, questions remain about cost

While Leqembi has been approved by the FDA, the drug will have to face other major hurdles before it can become widely available to patients.

Aduhelm, an Alzheimer’s drug also developed by Biogen and Eisai and approved by the FDA in 2021, failed to receive support from the Centers for Medicare and Medicaid Services even after it was approved by the FDA. As a result, Aduhelm has failed commercially and CMS only covers Aduhelm for patients in clinical trials.

A recent congressional investigation also revealed that Biogen knew the drug’s high price tag would make the drug more inaccessible to patients and strain the country’s Medicare budget.

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

Leqembi will be commercially available this month, but the vast majority of patients who could benefit from the drug in the U.S. will not have access to treatment until CMS decides to reverse its policy to cover Alzheimer’s drugs that target the amyloid-beta hypothesis. .

Cheung said the agency has made it clear in conversations with CMS that it needs full conventional approval from the FDA to consider lifting its current restrictions. He added that the company will request that the CMS review late-stage data in parallel with the FDA review to gain CMS coverage as soon as possible.

“Hopefully before the end of this year, we can have broader access to lecanemab from CMS,” he said.

Marc Goodman, an SVB Securities analyst, said in a December 18 note that there was “significant tension about the CMS repayment, which continues to be a surplus.”

Despite this, he expects 2023 to be “a transformative year for Alzheimer’s.”

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

This article has been updated with Leqembi price.

Read the original article on Business Insider

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