Mitsubishi Tanabe Pharma Submits New Drug Application forEdaravone to Treat ALS in the United States
Osaka, Japan, June 20, 2016 -- Mitsubishi Tanabe Pharma Corporation (MTPC) (HeadOffice: Osaka; President & Representative Director, CEO: Masayuki Mitsuka), today
announced that a New Drug Application has been submitted to the U.S. Food and Drug
Administration (FDA) for edaravone (MCI-186) for the treatment of amyotrophic lateralsclerosis (ALS), a rapidly progressive neurological disease. As many as 30,000 Americans
may be affected by ALS with more than 5,600 diagnosed annually.1This submission is the Company’s first step in accelerating its U.S. business development activities
as part of its “Medium-Term Management Plan 16-20: Open up the Future.” As part of this plan, theCompany established MT Pharma America, Inc., to commercialize approved pharmaceuticalproducts in the U.S. 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, eatand breathe.2,3 It is one of the most well-known neuromuscular diseases, affecting
approximately two in 100,000 people worldwide.4,5 While it is inherited in 5%–10% of cases,
the cause for the majority of cases is not well understood but may involve genetic andenvironmental factors.6,7 There is currently no cure.7“There is an urgent need for new treatment approaches that may affect the course of thisdevastating disorder,” said Lucie Bruijn, PhD, MBA, Chief Scientist, The ALS Association. “TheALS 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 inJapan. 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 foredaravone. It is not currently approved by the FDA for any use in the U.S.
〈〈For Details, Contact the Following Section〉〉
Corporate Communications Department
Tel +81-6-6205-5211

About Edaravone
Discovered by Mitsubishi Tanabe Pharma Corporation, edaravone is described as a freeradical scavenger that is believed to relieve the effects of oxidative stress, a likely factor in theonset and progression of ALS.4,8 Oxidative stress is thought to be an imbalance between theproduction of free radicals (unpaired, reactive electrons) and the ability of the body tocounteract or detoxify their harmful effects.9 In patients with ALS, there are consistentincreases in oxidative stress biomarkers.8

About MT Pharma America, Inc.
Based in Jersey City, NJ, MT Pharma America is a wholly-owned subsidiary of MTPC’s 100%owned U.S. holding company, Mitsubishi Tanabe Pharma Holdings America, Inc. MT Pharma
America is dedicated to delivering innovative solutions that address the unmet medical needsof patients in the United States. For more information, go to www.mt-pharma-america.com.
About Mitsubishi Tanabe Pharma Corporation
Mitsubishi Tanabe Pharma Corporation is a research-driven pharmaceutical company with a
Head Office based in Dosho-machi Osaka, the birthplace of Japan’s pharmaceutical industry.
As part of its “Medium-Term Management Plan 16-20: Open Up the Future,” the company isfocused on discovering drugs that address unmet medical needs in several priority disease
areas, including autoimmune disorders, diabetes, kidney and central nervous system diseases
and vaccines. Through this work, the company contributes to the healthier lives of people
around the world. For more information, go to www.mt-pharma.co.jp/e.

1

ALS Association. Quick Facts about ALS. http://www.alsa.org/news/media/quick-facts.html Accessed May 26, 2016.

2

The Mayo Clinic. Diseases and Conditions: Amyotrophic Lateral Sclerosis.
http://www.mayoclinic.org/diseases-conditions/amyotrophic-lateral-sclerosis/basics/causes/con-20024397. Accessed May 17, 2016.

3National Institute of Neurological Disorders and Stroke. Amyotrophic Lateral Sclerosis (ALS) Fact Sheet.
http://www.ninds.nih.gov/disorders/amyotrophiclateralsclerosis/detail_ALS.htm. Updated March 14, 2016. Accessed May 17, 2016.

4

Nagase M, Yamamoto Y, Miyazaki Y, et al. Increased oxidative stress in patients with amyotrophic lateral sclerosis and the effect of edaravone
administration. Redox Rep. 20155Chiò A, Logroscino G, Traynor B, et al. Global Epidemiology of Amyotrophic Lateral Sclerosis: a Systematic Review of the Published Literature.
Neuroepidemiology. 2013;41(2):118-130.

6

ALS Association. Familial Amyotrophic Lateral Sclerosis (FALS) and Genetic Testing. http://www.alsa.org/about-als/genetic-testing-for-als.html
Accessed June 8, 2016.

7Centers for Disease Control and Prevention. Prevalence of Amyotrophic Lateral Sclerosis — United States, 2010–2011.
http://www.cdc.gov/mmwr/preview/mmwrhtml/ss6307a1.htm. Accessed April 14, 2016.

8Manning, M.M. and Kelly-Worden, M. (2015) Potential Regulators of Sporadic ALS Development and Alternative Therapeutic Options.
Neuroscience & Medicine. 2015; 6, 5-12.

9Betteridge, D.J., What is oxidative stress? Metabolism. 2000;49: 3-8.

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