Traditional approval for the treatment of Alzheimer’s disease in the US
July 6, 2023: Eisai Co Ltd and Biogen Inc have announced that the US 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, making 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 generalisable to US 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 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. Full results of the Clarity AD study were presented at the Clinical Trials on Alzheimer’s Disease (CTAD) 2022 conference and simultaneously published in the peer-reviewed medical journal The New England Journal of Medicine on November 29, 2022.
Importantly, following 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.
“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 recognised and delivered to people living with this disease in the United States.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 maximise 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 humanised 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. In June 2023, the FDA’s Peripheral and Central Nervous System Drugs (PCNS) advisory committee voted unanimously that the data from Eisai’s Clarity AD clinical trial confirmed the clinical benefit of LEQEMBI for the treatment of AD. Committee members also confirmed the overall risk-benefit of LEQEMBI. On January 6, 2023, LEQEMBI was approved by the FDA under the accelerated approval pathway.
Eisai has developed and deployed Understanding ARIA™, a multi-faceted educational initiative to further advance understanding in the AD healthcare community of the real-world management and monitoring of amyloid-related imaging abnormalities (ARIA). In collaboration with experts in the field of medical imaging as well as major professional societies, Understanding ARIA™ offers resources and programmes that include peer-to-peer education, individual and group educational sessions and subject-matter-expert evaluation of historical case studies.
Eisai is committed to ensuring that appropriate patients have access to LEQEMBI and has established a Patient Assistance Programme to provide LEQEMBI at no cost, for eligible uninsured and underinsured patients, including Medicare beneficiaries, who meet financial need and other programme criteria. Additionally, Eisai offers patient support for improving access through LEQEMBI Patient Navigators, who will provide information about accessing LEQEMBI, help patients and their families understand their insurance coverage and options, and identify financial support programmes for eligible patients. People in the US can learn more about these services by visiting LEQEMBI.com, calling 1-833-4-LEQEMBI (1-833-453-7362), Monday-Friday, 8 a.m. to 8 p.m. Eastern Time or faxing an enrollment form to 1-833-770-7017.
Eisai serves as the lead of LEQEMBI development and regulatory submissions globally with both Eisai and Biogen co-commercialising and co-promoting the product and Eisai having final decision-making authority.
*Protofibrils are large Aβ aggregated soluble species of 75-5000 Kd.2,3.4