The FDA has accepted a new drug application (NDA) for edaravone (Mitsubishi Tanabe Pharma Corporation), an intravenous treatment for patients with amyotrophic lateral sclerosis (ALS). A decision on the application is expected in June 2017. If approved, the medication will be commercialized under the brand name Radicava through MT Pharma America, Inc.
Edaravone is a free radical scavenger that relieves the effects of oxidative stress––a 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. Patients with ALS show consistent increases in oxidative stress biomarkers.
ALS, also called Lou Gehrig’s disease, attacks nerve cells in the brain and spinal cord that are 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 ALS is inherited in 5% to 10% of cases, the cause of the majority of cases is not well understood but may involve genetic and environmental factors. There is currently no cure for the disease.
The NDA for edaravone was supported by a clinical research program in patients diagnosed with ALS in Japan. In 2015, edaravone was approved for use as a treatment for ALS in Japan and South Korea. In the same year, the FDA granted an orphan drug designation for edaravone. It is not currently approved for any use in the United States.
Source: Mitsubishi Tanabe Pharma; August 30, 2016.