Pharma Focus Asia

Mitsubishi Tanabe Pharma America Granted Orphan Drug Exclusivity for RADICAVA ORS® (edaravone) by FDA

Tuesday, April 09, 2024

Mitsubishi Tanabe Pharma America, Inc. (MTPA) has announced that the U.S. Food and Drug Administration (FDA) has granted seven years of Orphan Drug Exclusivity (ODE) for RADICAVA ORS® (edaravone). This recognition is based on the FDA's assessment of the oral form of edaravone as a significant advancement in patient care for individuals with amyotrophic lateral sclerosis (ALS), a rare disease affecting fewer than 200,000 people in the U.S. RADICAVA ORS, which previously received Priority Review and Fast Track designations from the FDA, offers a clinically superior option for patients due to its oral suspension route of administration, reducing the burden associated with intravenous administration.

The FDA's acknowledgment of ODE for RADICAVA ORS stems from its provision of a convenient and effective alternative to previously approved RADICAVA® (edaravone), administered intravenously. RADICAVA ORS can be self-administered orally or via feeding tube, eliminating the need for refrigeration or reconstitution before use. It comes in a portable bottle with an oral dosing syringe, making it more accessible for patients. According to healthcare providers, RADICAVA ORS requires only a few minutes for administration on treatment days and is best taken in the morning after fasting overnight.

Dr. Stanley H. Appel of the Houston Methodist Stanley H. Appel Department of Neurology expressed excitement about the FDA's recognition of ODE for RADICAVA ORS, emphasizing its significance in the ALS treatment landscape. This recognition, based on pivotal Phase 3 clinical trials, ensures a validated treatment option for several years.

The approval of RADICAVA® (edaravone) in the U.S. was grounded in the pivotal Phase 3 study (Study 19 or MCI186-19), which demonstrated its ability to slow the loss of physical function in ALS patients by 33 percent compared to placebo. RADICAVA ORS offers similar efficacy to RADICAVA in a more convenient oral formulation. Both formulations have been supported by robust clinical trial data and real-world evidence, with over 14,600 ALS patients treated in the U.S. to date.

RADICAVA and RADICAVA ORS are products of Mitsubishi Tanabe Pharma Corporation, researched and developed over a span of 13 years. RADICAVA was initially approved in Japan and South Korea in 2015 and has since received marketing authorizations in various countries, including the U.S., Canada, and Switzerland. RADICAVA ORS received FDA approval in 2022, and its regulatory approval has been granted in other countries as well. These formulations have been widely prescribed and used by ALS patients under the supervision of healthcare providers globally.



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