A US Food and Drug Administration (FDA) advisory panel unanimously backed the Alzheimer’s drug Leqembi last week, setting it up for full approval.
FDA advisory panel documents are now posted for $BIIB’s Leqembihttps://t.co/cnqozDBAC6
— FDAadcomms (@FDAadcomms) June 7, 2023
The Path to Approval
In January, the FDA granted accelerated approval for lecanemab, marketed under the name Leqembi. The anti-amyloid treatment showed promise in slowing the progression of cognitive decline in patients in the early stages of Alzheimer’s disease.
The drug met both the primary and all key secondary endpoints for the target patient population in the phase 3 data from the Clarity AD global clinical trial. Results from the study, which were statistically significant, were published in the New England Journal of Medicine.
“The FDA Advisory Committee’s support for traditional approval of Leqembi for people with early Alzheimer’s is welcomed and celebrated by the Alzheimer’s Association,” said Joanne Pike, president and CEO of the Alzheimer’s Association in a statement. “With this action, we are one step closer to enabling more people living with Alzheimer’s to retain more of their capabilities intact, allowing them to do the things they want to do and love to do best.”
We welcome and celebrate the @US_FDA Advisory Committee’s unanimous endorsement of the efficacy and clinical benefit of lecanemab (Leqembi) as part of the treatment’s traditional approval process. Read our statement: https://t.co/7wnRcqWoYS. pic.twitter.com/5jlywXVgPU
— Alzheimer's Association (@alzassociation) June 9, 2023
Cautions, Next Steps
However, the drug does increase the risk of swelling and bleeding in the brain. The current prescriber documentation emphasizes the need for real-world evidence and long-term safety data collection. Eisai, the maker of the drug, explicitly states that the drug is suitable only for patients in the early stages of the neurodegenerative disease. The company recommends testing patients before administering the drug to determine if there is a buildup of amyloid proteins in the brain.
Despite these caveats, Pike stated that the association fully agrees with the FDA advisory committee. She says that Leqembi’s clinical benefits that outweigh its risks. In a category still light on options, Pike emphasized that Americans living with Alzheimer’s disease “deserve access to FDA-approved therapies without barriers, similar to people with cancer, heart disease, and HIV/AIDS.”
The FDA has granted Priority Review to advance Leqembi to the next phase of traditional approval application, which involves a full FDA review. For the treatment to receive full agency approval, Eisai must complete this action by July 6, 2023. The company has not yet released proposed pricing information.
Live-blogging the FDA advisory panel on an Alzheimer's treatment https://t.co/4YpIBJhehw
— Matthew Herper (@matthewherper) June 9, 2023
Looking to the Future
More than six million Americans of all ages live with Alzheimer’s. The condition has no cure. Current treatments offer limited improvement for symptoms such as memory deterioration and loss of function.
The FDA Peripheral and Central Nervous System Drugs Advisory Committee, which voted on the drug, expressed the goal of delivering multiple therapies for an individualized approach in Alzheimer’s.
“In today’s pipeline, over three quarters of drugs in clinical development target novel pathways, priming the field for the next generation of drugs that address a variety of underlying factors,” said Howard Fillit, MD, co-founder, and chief science officer of the committee.
