Eisai Co., Ltd. and Biogen Inc. have announced that the U.S. Food and Drug Administration (FDA) has approved the supplemental Biologics License Application (sBLA) supporting the traditional approval of LEQEMBI (lecanemab-irmb) 100 mg/mL injection for intravenous use.
The decision makes LEQEMBI the first and only approved treatment shown to reduce the rate of disease progression and to slow cognitive and functional decline in adults with Alzheimer’s disease (AD).
LEQEMBI demonstrated clinically meaningful slowing of cognitive and functional decline in a patient group generalizable to U.S. Medicare beneficiaries, which included a mix of racial and ethnic groups, patients with common comorbid conditions, concomitant medications and patients with mild cognitive impairment (MCI) due to AD or mild AD.
Treatment with LEQEMBI should be initiated in patients with MCI or mild dementia stage of disease, (collectively referred to as early AD) the population in which treatment was initiated in clinical trials.
LEQEMBI‘s trial data
LEQEMBI’s traditional approval is based on phase 3 data from Eisai’s large, global Clarity AD clinical trial, in which LEQEMBI met its primary endpoint and all key secondary endpoints with statistically significant results and confirmed the clinical benefit of LEQEMBI. The primary endpoint was the global cognitive and functional scale, Clinical Dementia Rating Sum of Boxes (CDR-SB). LEQEMBI treatment reduced clinical decline on CDR-SB by 27% at 18 months compared to placebo. Additionally, the secondary endpoint of AD Cooperative Study-Activities of Daily Living Scale for Mild Cognitive Impairment (ADCS MCI-ADL), as measured by people caring for patients with AD, noted a statistically significant benefit of 37%. This measures the ability of patients to function independently, including being able to dress, feed themselves and participate in community activities.
Following the FDA’s traditional approval of LEQEMBI, CMS confirmed that broader coverage of LEQEMBI is now available and released more details on the registry, including the easy-to-use data submission process. The CMS-facilitated registry is now available for healthcare professionals to submit required patient data to CMS. Eisai said it is pleased that Medicare will cover the therapy for appropriate patients. This will facilitate reimbursement for and access to LEQEMBI across a broad range of heathcare settings in the U.S.
“Today, the FDA approved LEQEMBI under the traditional approval pathway, making LEQEMBI the first and only approved anti-amyloid Alzheimer’s disease treatment shown to reduce the rate of disease progression and to slow cognitive impairment in the early and mild dementia stages of the disease. As a research and development-focused company based on our hhc (human health care) concept, we are proud that the results of Eisai’s AD research over the past 40 years have been recognized and delivered to people living with this disease in the United States,” said Haruo Naito, chief executive officer at Eisai.
“Alzheimer’s disease is a progressive, fatal disease that greatly impacts not only the people living with it, but also their loved ones, care partners and society. We continue to work to create broad and simple access to LEQEMBI for patients and to support diagnosis and treatment at the early stage of the disease. Eisai will diligently work to educate physicians on the safe and appropriate use of LEQEMBI to maximize its benefit to people living with early AD and their families.”
“Today marks a breakthrough in the treatment of Alzheimer’s disease, and we are proud to be at the forefront of ushering in a new era of advances for a disease that was previously considered untreatable. We would like to express our sincere appreciation to those who have worked tirelessly to find a treatment for this unrelenting disease, without whom this progress would not be possible,” said Christopher A. Viehbacher, president and chief executive officer of Biogen.
“Our focus is now on the path forward, working alongside Eisai with the goal of making LEQEMBI accessible to eligible patients as soon as possible.”
LEQEMBI is a humanized immunoglobulin gamma 1 (IgG1) monoclonal antibody directed against aggregated soluble (protofibril) and insoluble forms of amyloid beta (Aβ). Critically, LEQEMBI targets and clears the most neurotoxic form of Aβ that continuously accumulates as well as removes the existing plaques to treat this progressive, chronic disease.
The Alzheimer’s Association praised the announcement.
“This treatment, while not a cure, gives people in the early stages of Alzheimer’s disease more time to maintain their independence and do the things they love,” said Joanne Pike, Alzheimer’s Association president and CEO.
“While we continue efforts to discover new targets and test new treatments, people living with this fatal disease deserve the opportunity to discuss and make the choice with their doctor if an FDA-approved treatment is right for them.”
This is a momentous day for people living with early-stage Alzheimer’s and their families,” said Hugh Courtney, 60, of Concord, Mass., who was diagnosed with Alzheimer’s disease in 2020.
“I was in the Leqembi clinical trial, so I am really excited about today’s FDA approval. It gives others living with early-stage Alzheimer’s a chance to pursue treatment that may slow progression of their disease.”
The Alzheimer’s Drug Discovery Foundation (ADDF) also welcomed the news.
“This is encouraging news, and more importantly, the approval of Leqembi will serve as a catalyst for driving further developments and investments in the Alzheimer’s pipeline,” said Howard Fillit, co-founder and chief science officer of the ADDF.
“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.”