In the 48-week study, a 24-week double blind placebo controlled phase was followed by a 24-week open label active treatment phase. Data previously presented at the 2016 Annual Meeting of the American Academy of Neurology, showed edaravone met the primary efficacy endpoint of mean change in the ALSFRS-R at 24 weeks. The frequency of serious adverse events was similar to placebo.
"These findings suggest that intervention with edaravone may provide a treatment option to people living with ALS when therapy is promptly initiated," said Joseph M. Palumbo, MD, Vice President, Medical Sciences and Translational Research, Mitsubishi Tanabe Pharma Development America, Inc.
Discovered by Mitsubishi Tanabe Pharma Corporation (MTPC), edaravone is described as a free radical scavenger that is believed to relieve the effects of oxidative stress, a likely factor in the onset and progression of ALS. Oxidative stress is thought to be an imbalance between the production of free radicals and the ability of the body to counteract or detoxify their harmful effects. In patients with ALS, there are consistent increases in oxidative stress biomarkers.