US regulators have agreed to review Mitsubishi Tanabe's application to market edaravone for the treatment of amyotrophic lateral sclerosis (ALS), a rapidly progressive neurological disease.
As many as 30,000 Americans may be affected by ALS, also referred to as Lou Gehrig's disease, a neuromuscular condition that attacks nerve cells in the brain and spinal cord responsible for controlling voluntary muscles, such as those needed to move, speak, eat and breathe.
The condition is inherited in 5 percent to 10 percent of cases, but the cause for the majority of cases is not well understood, and there is currently no cure.
Edaravone (MCI-186) 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.
The drug has already been granted Orphan Drug status in the US, and its filing is supported by the same data that secured its regulatory approval in Japan and South Korea last year.
The Phase III trial, presented earlier this year at the American Academy of Neurology annual meeting, met its primary endpoint of mean change in the ALS Functional Rating Scale-Revised at 24 weeks versus the standard of care.
"There is an urgent need for new treatment approaches that may affect the course of this devastating disorder. The ALS Association is encouraged by the efforts of Mitsubishi Tanabe Pharma to make this drug available to people living with ALS in the US," noted the group's chief scientist Lucie Bruijn.