Eisai Initiates Preparations for New Drug Application in Japan for Ultrahigh-Dose Methylcobalamin in ALS Based on JETALS Trial Results

May 11, 2022 10:51 CST Updated 10:51

Eisai

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Tokushima University

Tokushima University, abbreviated as “Toku Dai,” is one of Japan’s national universities. It was established in 1949 through the merger of Tokushima Normal School, Tokushima Youth Normal School, Tokushima Medical College, Tokushima Specialist School of Medicine, Tokushima High School, and Tokushima Technical College. The university’s foundational philosophy emphasizes respect for individual student characteristics and aims to cultivate professionals who possess practical skills, a proactive spirit, and an international outlook.

TokyoMay 11, 2022/PR Newswire/ -- Eisai Co., Ltd. (Headquarters: Tokyo, CEO: Haruo Naito, "Eisai") announced that in Japan, the company has begun preparations for a new drug application for ultra-high dose mecobalamin (development code: E0302) for the treatment of amyotrophic lateral sclerosis (ALS). This application is based on favorable clinical results from JETALS (Japan Early Trial of Ultra-High Dose Mecobalamin for ALS: JETALS), a Phase III investigator-initiated trial to evaluate the efficacy and safety of ultra-high dose mecobalamin (mecobalamin) in patients with early-onset ALS. The paper was published by a research team led by Distinguished Professor Ryuji Kaji (Principal Investigator) and Professor Yuishin Izumi (Coordinating Investigator) from the Department of Neurology at the Graduate School of Biomedical Sciences, Tokushima University. Eisai submitted an orphan drug designation application for the drug to Japan's Ministry of Health, Labour and Welfare (MHLW) in March 2022 and plans to submit a new drug application in fiscal year 2023.

For ultra-high-dose methylcobalamin, Eisai submitted a new drug application for the treatment of ALS in May 2015 based on the results of the ALS II/III clinical trial (Study 761). However, the application was withdrawn in March 2016 after receiving feedback from the Pharmaceuticals and Medical Devices Agency (PMDA) indicating insufficient application materials.

However, another analysis of Study 761 indicated that ultra-high-dose mecobalamin extended survival and slowed disease progression in ALS patients who began treatment within 12 months after the onset of ALS. To re-examine its efficacy and safety within one year after the onset of ALS, a research team led by Tokushima University initiated the JETALS clinical trial with support from the Japan Agency for Medical Research and Development (AMED). The study demonstrated the efficacy, safety, and tolerability of ultra-high-dose mecobalamin, and the results have now been published in the peer-reviewed journal JAMA Neurology. Based on the results of JETALS and following consultation with Tokushima University, Eisai has decided to resubmit the approval application for ultra-high-dose mecobalamin for ALS in Japan.

Eisai focuses on neuroscience as a key therapeutic area and is committed to the development of new drugs in this field to address unmet medical needs and further contribute to the benefits of patients and their families.