April 9th, 2024

RADICAVA ORS® as Orphan drug designation and approval in US
Mitsubishi Tanabe Pharma Corporation (Head Office: Chuo-ku, Osaka; Representative
Director: Akihiro Tsujimura; hereinafter, "MTPC"), a member of the Mitsubishi ChemicalHoldings Group, announced that on March 28, 2024, the United States Food and Drug
Administration (FDA) has recognized seven years of orphan-drug exclusive approval for
RADICAVA ORS® (edaravone) for treatment of amyotrophic lateral sclerosis (ALS). The
period of exclusive approval is seven years from May 12, 2022, the date of approval of New
Drug Application (NDA).
ALS is an idiopathic neurodegenerative disease in which motor neurons selectively
degenerate and vanish. Muscle strength declines throughout the entire body, including thelimb, facial, and respiratory muscles, and muscular atrophy progress. While the cause forthe majority of cases is not well understood but may involve genetic and environmental
factors. It is one of the most well-known neuromuscular diseases, affecting approximately
two in 100,000 people worldwide.
RADICAVA ORS® is an oral suspension formulation that contains the same active
ingredient as RADICAVA® for intravenous infusion. MTPC Group has been developing
RADICAVA ORS® as a new treatment option for ALS patients in order to address unmet
needs of people living with ALS. Promoting RADICAVA ORS® in the U.S. is handled by
Mitsubishi Tanabe Pharma America, Inc.
MTPC group will continue working to contribute to people living with ALS worldwide as aleading company in the treatment of ALS by delivering RADICAVA ORS® and RADICAVA®.

Contact:
Mitsubishi Chemical Group Corporation
Communications Division
Osaka Communications Department+81-6-6205-5119

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■About amyotrophic lateral sclerosis: ALS
ALS is an idiopathic neurodegenerative disease in which motor neurons selectivelydegenerate and vanish. Muscle strength declines throughout the entire body, including theextremity, facial, and respiratory muscles, and muscular atrophy progresses. While thecause for the majority of cases is not well understood but may involve genetic andenvironmental factors. It is one of the most well-known neuromuscular diseases andincidence is approximately two in 100,000 people per year worldwide.
■About RADICAVA ORS®
RADICAVA ORS® is specifically formulated for patients with ALS and provides a flexible
administration option with a 5ml dose taken orally or via feeding tube. One cycle ofadministration of RADICAVA ORS® is a total of 28 days, including both the dosing period
and the drug-free period, and that cycle is repeated. The first cycle has a dosing period of
14 consecutive days of administration followed by a drug-free period of 14 days. In
subsequent cycles, patients receive daily dosing for 10 days within a 14-day dosing period,followed by a 14-day drug-free period. RADICAVA ORS® are available in bottles and do not
require water or solution to administer. RADICAVA ORS® needs to be refrigerated during
the distribution process and in pharmacies, but patients can keep it at room temperature.
■About Edaravone
Edaravone is a free radical scavenger discovered by MTPC. It was approved by theMinistry of Health, Labour and Welfare in April 2001 for the treatment of patients with acuteischemic stroke and is marketed in Japan under the product name of Radicut®. Edaravone
has the effect of scavenging free radicals that arise accompanying cerebral ischemia,
controlling the lipid peroxidation reaction, and protecting neurons in the region of theischemia and the surrounding region. Accordingly, it is thought that edaravone has theeffect of scavenging free radicals, which increase in ALS, protecting motor neurons fromoxidative stress, and delaying the decline in muscle strength and the progress of muscularatrophy.
This product obtained approval for an additional indication of ALS in Japan in 2015 and hassince been launched in 9 countries (South Korea, the U.S., Canada, Switzerland, Indonesia,Thailand, Malaysia and Brazil).
■About Mitsubishi Tanabe Pharma America, Inc.
Based in Jersey City, N.J., Mitsubishi Tanabe Pharma America, Inc. (MTPA) is a whollyowned subsidiary of Mitsubishi Tanabe Pharma Corporation (MTPC). It was established by
MTPC in 2015 to develop and advance our pipeline as well as commercialize approved
pharmaceutical products in North America. For more information, please visit www.mtpharma-america.com
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