Mitsubishi Tanabe Pharma Corporation (Head Office: Osaka; President & Representative Director, CEO: Dr. Masayuki Mitsuka) has presented data that show patients with amyotrophic lateral sclerosis (ALS) given edaravone intravenously in 10-14 day cycles for 48 weeks experienced significantly less functional loss as measured by the ALS Functional Rating Scale-Revised (ALSFRS-R). The data were presented at the 27th International Symposium on ALS/MND in Dublin, Ireland.
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.
ALS, sometimes called Lou Gehrig's disease, attacks the nerve cells in the brain and the spinal cord responsible for controlling voluntary muscles, such as those needed to move, speak, eat and breathe. It is one of the most well-known neuromuscular diseases, affecting approximately two in 100,000 people worldwide. While it is inherited in 5% - 10% of cases, the cause for the majority of cases is not well understood but may involve genetic and environmental factors. There is currently no cure.
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.
In 2015, edaravone was approved for use as a treatment for ALS in Japan and South Korea. In the same year, the FDA and the European Commission granted Orphan Drug Designation for edaravone. It is not currently approved by the FDA for any use in the U.S.