FDA Accepts Mitsubishi Tanabe Pharma’s NDA Filingfor Edaravone to Treat ALS••Edaravone may be the first ALS treatment approved in U.S. in more than 20 years.
As many as 30,000 Americans are estimated to be affected by ALS and more than 5,600
are diagnosed annually.1Osaka, Japan, August 30, 2016 -- Mitsubishi Tanabe Pharma Corporation (MTPC) (HeadOffice: Osaka; President & Representative Director, CEO: Masayuki Mitsuka), today
announced that the U.S. Food and Drug Administration (FDA) has accepted the company’sNew Drug Application (NDA) for edaravone (MCI-186) an intravenous treatment foramyotrophic lateral sclerosis (ALS), a rapidly progressive neurological disease. A decision on
the application is anticipated on June 16, 2017 based on the Prescription Drug User Fee Act. If
approved, the medicine will be commercialized, under the brand name RADICAVA™, throughthe newly-formed MT Pharma America, Inc.
“This is an important milestone for Mitsubishi Tanabe Pharma and for the U.S. ALS
community,” said Joseph M. Palumbo, MD, Vice President, Head of Clinical Research,
Mitsubishi Tanabe Pharma Development America, Inc. “There is an urgent need for new
treatment options in ALS and we are now an important step closer to potentially making that areality. We look forward to working with the FDA as part of the review process.”
About ALS
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.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.6
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.3,8 Oxidative stress is thought to be an imbalance between theproduction of free radicals and the ability of the body to counteract or detoxify their harmful
effects.9 In patients with ALS, there are consistent increases in oxidative stress biomarkers.7

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. It was established by MTPC to commercialize approved
pharmaceutical products in the U.S. with plans to expand its product line through collaborations
with partners. 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 central nervous system diseases, autoimmune disorders, diabetes, kidneydisease and vaccines. Through this work, the company contributes to the healthier lives ofpeople 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.

2The 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.

3

National 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.

4Nagase M, Yamamoto Y, Miyazaki Y, et al. Increased oxidative stress in patients with amyotrophic lateral sclerosis and the effect of edaravoneadministration. 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.

7

Centers 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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