Updated on 21 June 2016
Singapore: Mitsubishi Tanabe Pharma Corporation has announced that a New Drug Application has been submitted to the US Food and Drug Administration (FDA) for edaravone (MCI-186) for the treatment of amyotrophic lateral sclerosis (ALS), a rapidly progressive neurological disease. As many as 30,000 Americans may be affected by ALS with more than 5,600 diagnosed annually.
This submission is the company's first step in accelerating its US business development activities as part of its ‘Medium-Term Management Plan 16-20: Open up the Future'. As part of this plan, the company established MT Pharma America Inc., to commercialize approve pharmaceutical products in the US with plans to expand its product line through collaborations with partners.
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, says Mitsubishi Tanabe.
"There is an urgent need for new treatment approaches that may affect the course of this devastating disorder," said Lucie Bruijn, PhD, MBA, Chief Scientist, The ALS Association. "The ALS Association is encouraged by the efforts of Mitsubishi Tanabe Pharma to make this drug available to people living with ALS in the U.S."
The edaravone NDA is 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 and the European Commission granted Orphan Drug Designation for edaravone. It is not currently approved by the FDA for any use in the US.