Eisai gets Japanese orphan drug designation for ultrahigh-dose mecobalamin with prospective indication for functional impairment of amyotrophic lateral sclerosis
ALS is an intractable, progressive, neurodegenerative disease that results in severe muscle atrophy and weakness in the muscles due to motor neuron dysfunction.
Japan: Eisai Co., Ltd. has announced that the company has received orphan drug designation for ultrahigh-dose mecobalamin, with a prospective indication for delaying the progression of disease and functional impairment of amyotrophic lateral sclerosis (ALS), by the Ministry of Health, Labour and Welfare (MHLW).
Basd on a favorable clinical results of the JETALS (The Japan Early-Stage Trial of Ultrahigh-Dose Methylcobalamin for ALS), an investigator-initiated Phase III trial to evaluate efficacy and safety of ultrahigh-dose methylcobalamin (mecobalamin) in early onset ALS patients, led by a research team with Extraordinary Professor Ryuji Kaji (Principal Investigator), Tokushima University and Professor Yuishin Izumi (Coordinating Investigator), the Department of Neurology, Tokushima University Graduate School of Biomedical Sciences, Eisai has initiated preparation on a new drug application (NDA) of ultrahigh-dose mecobalamin for ALS, and plans to submit a NDA during the fiscal year 2023 in Japan.
"Eisai considers neurology a therapeutic area of focus and is committed to new drug development in this field in order to fulfill unmet medical needs, and ultimately further its contribution to improving the benefit of patients and their families," the company stated.
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According to the number of patients issued a Certificate of the Recipient of Specified Disease Treatment, there were 10,514 patients with ALS in Japan in 2020. Currently, there is no curative treatment established for ALS, and since there are only limited number of medicines approved in Japan and abroad, this is a disease with significant unmet medical needs.
