The Food and Drug Administration on Thursday granted full approval for the first time to an experimental Alzheimer's drug, triggering a process that could expand coverage of the $26,500 treatment to millions of patients.
Why it matters: Eisai and Biogen's Leqembi was found to have modest success slowing the disease's progression in clinical trials, by delaying cognitive decline by 27% over 18 months.
- The approval came despite safety concerns following three patient deaths during clinical trials.
- "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," said Teresa Buracchio, acting director for the FDA’s office of neuroscience.
- "This confirmatory study verified that it is a safe and effective treatment for patients with Alzheimer’s disease," Buracchio added.
- The agency has until now given accelerated approvals to Alzheimer's treatments, meaning drugmakers could bring them to market based on preliminary evidence while they carried out confirmatory trials that could take years to complete.
The big picture: The FDA's action sets the stage for the Centers for Medicare and Medicaid Services to expand coverage for drugs that slow Alzheimer's progression. Prior to FDA's approval, the agency restricted coverage to patients enrolled in clinical trials.
- CMS announced in June that it would revise that policy as soon as a drug of that kind received traditional approval and pay for the drug for Medicare beneficiaries if their health providers agree to collect and share data about the drug's performance.
- The agency said that data registries will "adhere to robust privacy protections," while still allowing researchers to access the information to conduct studies around how the drug works in real life.
- "With FDA's decision, CMS will cover this medication broadly while continuing to gather data that will help us understand how the drug works. This is welcome news for the millions of people in this country and their families who are affected by this debilitating disease," said CMS Administrator Chiquita Brooks-LaSure.
What they're saying: Patient advocacy groups and research organizations celebrated the FDA's approval.
- "Leqembi's traditional approval is a ray of hope for millions of patients who are doing everything in they can to enhance and extend their lives and reduce their families' burdens," said George Vradenburg, chairman and co-founder of the patient advocacy group UsAgainstAlzheimer's.
- "This treatment, while not a cure, can give people in the early stages of Alzheimer's more time to maintain their independence and do the things they love," said Joanne Pike, CEO of the Alzheimer's Association.
- "We finally have clarity around amyloid’s modest effect on cognitive decline. Now, it is more important than ever to double down and widen our focus to developing the next generation of drugs based on the biology of aging that can lead to a combination therapy and precision medicine approach," said Howard Fillit, co-founder and chief science officer of the Alzheimer’s Drug Discovery Foundation.
By the numbers: The Institute for Clinical and Economic Review said in April that, with its $26,500 price tag, Leqembi would cause "rapid growth in health care insurance costs that would threaten sustainable access to high-value care for all patients."
- The organization suggested that the drug should cost between $8,900 and $21,500.
Don't forget: Over 6 million people are living with Alzheimer's in the U.S., and the number is expected to more than double by 2050, according to the Alzheimer's Association.
Catch up quick: Last month, independent FDA advisers unanimously said that Leqembi had clinical benefit for treating Alzheimer's.
- Some advisers reiterated that since there are some risks associated with Leqembi, that patients should decide with their physicians if taking the drug is right for them.
