Eisai initiates NDA in Japan for ultrahigh-dose mecobalamin based on results of JETALS clinical trial for amyotrophic lateral sclerosis.

Eisai submitted an application for an orphan drug designation of this agent to the Ministry of Health, Labour and Welfare (MHLW) in March 2022, and plans to submit a new drug application in fiscal year 2023.

For ultrahigh-dose mecobalamin, Eisai submitted a new drug application as treatment for ALS in May 2015 based on the results of a Phase II/III clinical trial (Study 761) for ALS, but withdrew the application in March 2016 after receiving the opinion about the application package not being sufficient from the Pharmaceuticals and Medical Devices Agency (PMDA). However, an additional analysis of Study 761 suggested that ultrahigh-dose mecobalamin prolonged survival period and suppressed progression in ALS patients who commenced treatment within 12 months of ALS onset.

In order to re-examine its efficacy and safety in ALS patients within 1 year of onset, JETALS was conducted as an investigator-initiated clinical trial by the research team led by the Tokushima University with the support of the Japan Agency for Medical Research and Development (AMED). The results of the study showed the efficacy, safety, and tolerability of ultrahigh-dose mecobalamin, which have now been published in the peer-reviewed journal JAMA Neurology. Based on the results of JETALS, in consultation with the Tokushima University, Eisai has decided to newly submit the application for approval of ultrahigh-dose mecobalamin for ALS in Japan.

See- JAMA Neurol. 2022; doi: 10.1001/jamaneurol.2022.0901.May 9, 2022 "Efficacy and Safety of Ultrahigh-Dose Methylcobalamin in Early-Stage Amyotrophic Lateral Sclerosis: A Randomized Clinical Trial".-Ryosuke Oki, MD; Yuishin Izumi, MD, PhD; Koji Fujita, MD, PhD; Ryosuke Miyamoto, MD; Hiroyuki Nodera, MD, PhD. et al.