Eisai Submits New Drug Application in Japan for Ultra-High-Dose Mecobalamin to Treat Amyotrophic Lateral Sclerosis

TokyoFebruary 2, 2024PR Newswire -- Eisai Co., Ltd. submitted a New Drug Application (NDA) for ultra-high dose mecobalamin (development code: E0302) to Japan's Pharmaceuticals and Medical Devices Agency (PDMA) on January 26 for the treatment of Amyotrophic Lateral Sclerosis (ALS). Ultra-high dose mecobalamin received orphan drug designation from Japan's Ministry of Health, Labour and Welfare (MHLW) in May 2022.

This application is based on the research group led by Tatsuo Kaji, Special Professor at Tokushima University (Principal Investigator), Yukihiro Izumi, Professor of Neurology at the Graduate School of Biomedical Sciences, Tokushima University (Coordinating Investigator), and Satoshi Kuwabara, Professor of Neurology at the Graduate School of Medicine, Chiba University (Coordinating Investigator). It aims to evaluate the efficacy and safety of ultra-high-dose mecobalamin in early-stage ALS patients and is proposed on the basis of the Phase III trial results of JETALS (Japan Early Trial of Ultra-High-Dose Mecobalamin for ALS). The findings of JETALS have been published in the peer-reviewed journal JAMA Neurology.

ALS is a refractory and progressive neurodegenerative disease characterized by severe muscle atrophy and weakness caused by motor neuron dysfunction. The primary cause of death in this condition is respiratory failure due to respiratory muscle paralysis, and without the use of a ventilator, patients typically die within approximately 3 to 6 years after onset.Patients in JapanThe number of patients is approximately 10,000. Currently, there is no effective treatment for ALS, and the number of drugs approved in Japan and other countries is limited, so the medical needs for this disease remain unmet.

Eisai regards neurology as one of its key therapeutic areas. As an "hhc" company (human health care), Eisai is committed to addressing unmet medical needs in the field of neurology, contributing to the well-being of patients and people in their daily lives.