Eisai Receives Orphan Drug Designation in Japan for High-Dose Mecobalamin to Treat Amyotrophic Lateral Sclerosis

Expected Indication: To delay the disease progression and functional impairment of amyotrophic lateral sclerosis.

TokyoMay 30, 2022PR Newswire -- Eisai Co., Ltd. (Headquarters: Tokyo, CEO: Haruo Naito, "Eisai") announced that its ultra-high dose formulation of mecobalamin has been granted orphan drug designation by Japan's Ministry of Health, Labour and Welfare for the anticipated indication of delaying disease progression and functional impairment in amyotrophic lateral sclerosis (ALS).

ALS is an intractable, progressive, neurodegenerative disease with significant unmet medical needs. In Japan, the estimated number of ALS patients is approximately 10,000.

Based on the positive clinical results of JETALS (Japan Early Trial of Ultra-high Dose Mecobalamin for ALS), Eisai has begun preparations for a New Drug Application (NDA) for ultra-high dose mecobalamin in the treatment of ALS and plans to submit the NDA in Japan during the fiscal year 2023. The project is led by a research team consisting of Special Appointed Professor Ryuji Kaji (Principal Investigator) from Tokushima University and Professor Yuishin Izumi (Coordinating Investigator) from the Department of Neurology at the Graduate School of Biomedical Sciences, Tokushima University. It is an investigator-initiated Phase III trial aimed at evaluating the efficacy and safety of ultra-high dose mecobalamin in patients with early-onset ALS.

Eisai regards neuroscience as a key therapeutic area and is committed to the research and development of new drugs in this field to address unmet medical needs, ultimately contributing further to the well-being of patients and their families.