SYNH Syneos Health Inc

Orphan Drug Exclusivity recognized for RADICAVA ORS based on major contribution to patient care 

JERSEY CITY, N.J., April 8, 2024 /PRNewswire/ -- Mitsubishi Tanabe Pharma America, Inc. (MTPA) today announced that the U.S. Food and Drug Administration (FDA) has recognized seven years of Orphan Drug Exclusivity (ODE) for RADICAVA ORS^® (edaravone) based on their assessment that the oral form of edaravone constitutes a major contribution to patient care for people living with amyotrophic lateral sclerosis (ALS). The FDA's Orphan Drug program is designed to support the development of drugs that treat rare diseases which affect less than 200,000 people in the U.S. Previously, the FDA granted RADICAVA ORS Priority Review and Fast Track designations.

The FDA recognized ODE for RADICAVA ORS because it provides a clinically superior option for patients due to its oral suspension route of administration that can help reduce the burden patients face with intravenous (IV) administration of previously approved RADICAVA^® (edaravone). RADICAVA ORS is self-administered, can be taken orally or via feeding tube, comes in a portable bottle with an oral dosing syringe and no need for patients to refrigerate or reconstitute before taking. With appropriate instruction from a healthcare provider (HCP), RADICAVA ORS may take only a few minutes to administer on treatment days and should be taken in the morning after overnight fasting.¹

"I'm thrilled at the FDA's recognition of ODE to RADICAVA ORS," said Stanley H. Appel, MD, Houston Methodist Stanley H. Appel Department of Neurology. "This comes at a critical time in the ALS treatment landscape and helps ensure that our patients and HCPs have a treatment option validated for several years, including by the pivotal Phase 3 clinical trial."

The U.S. approval of RADICAVA^® (edaravone) was based on the pivotal Phase 3 study (Study 19 or MCI186-19), in which RADICAVA was shown to slow the loss of physical function by 33 percent in 68 patients vs. placebo (n=66 patients), measured over a 24-week time period by the ALS Functional Rating Scale-Revised (ALSFRS-R).¹ On average, patients on RADICAVA lost 2.49 fewer points on the ALSFRS-R vs. those in the placebo arm (p=0.0013).^1,2 RADICAVA ORS offers the same efficacy as RADICAVA in a different formulation.^1,2 The safety profile of RADICAVA was demonstrated in pooled placebo-controlled trials in which 184 patients with ALS were administered RADICAVA (60 mg) in 24-week treatment cycles.¹ The safety profile of RADICAVA ORS was demonstrated in a 6-month, Phase 3, open-label clinical trial in 185 patients. Please see Important Safety Information below and Full Prescribing Information here.

"We recognize the profound impact that an oral formulation has on the ALS community, in part by offering a less burdensome option for people living with ALS," said Calaneet Balas, President & CEO, ALS Association. "We thank Mitsubishi Tanabe Pharma America for continuing their efforts to help provide treatments to people living with ALS."

To date, in the U.S., over 14,600 people living with ALS have been treated with RADICAVA and RADICAVA ORS and has been prescribed by over 2,300 HCPs.^3,4 Both formulations are supported by a robust set of data from multiple clinical trials, post-hoc analysis and real-world evidence (RWE) studies evaluating the efficacy and safety. These results have been published in over 40 peer-reviewed articles.

About RADICAVA^® (edaravone) and RADICAVA ORS^® (edaravone)
The U.S. Food and Drug Administration (FDA) approved RADICAVA^® (edaravone) on May 5, 2017, and the oral formulation RADICAVA ORS^® (edaravone) on May 12, 2022, for the treatment of amyotrophic lateral sclerosis (ALS). In 2024, the FDA recognized RADICAVA ORS with Orphan Drug Exclusivity based on the major contribution to patient care of the innovative oral formulation. RADICAVA is administered in 28-day cycles by intravenous (IV) infusion. It takes 60 minutes to receive each 60 mg dose. For the initial cycle, the treatment is infused daily for 14 consecutive days, followed by a two-week drug-free period. All cycles thereafter are infused daily for 10 days within a 14-day period, followed by a two-week drug-free period. RADICAVA ORS is taken daily for 14 consecutive days followed by a 14-day drug-free period for the initial treatment cycle. For subsequent treatment cycles, RADICAVA ORS is taken for 10 days within a 14-day period followed by a 14-day drug-free period. RADICAVA ORS should be taken in the morning after overnight fasting. Patients should not eat or drink (except water) within one hour after taking RADICAVA ORS.¹ 

Edaravone was discovered and developed for ALS by Mitsubishi Tanabe Pharma Corporation (MTPC) and commercialized in the U.S. by Mitsubishi Tanabe Pharma America, Inc. (MTPA). The MTPC group companies began researching ALS in 2001 through an iterative clinical platform over a 13-year period. In 2015, edaravone was approved as RADICUT^® for the treatment of ALS in Japan and South Korea. Marketing authorizations were subsequently granted in Canada (October 2018), Switzerland (January 2019), Indonesia (July 2020), Thailand (April 2021), Malaysia (December 2021) and Brazil (February 2024). Marketing authorization for RADICAVA^® Oral Suspension was granted in Canada (November 2022) and Switzerland (May 2023), and RADICUT^® Oral Suspension 2.1% was granted regulatory approval in Japan in December 2022. To date, in the U.S., RADICAVA and RADICAVA ORS have been used to treat over 14,600 people with ALS, with over 1.8-million days of therapy, and have been prescribed by over 2,300 HCPs.^3-5

IMPORTANT SAFETY INFORMATION

Hypersensitivity Reactions
RADICAVA (edaravone) and RADICAVA ORS (edaravone) are contraindicated in patients with a history of hypersensitivity to edaravone or any of the inactive ingredients of this product. Hypersensitivity reactions (redness, wheals, and erythema multiforme) and cases of anaphylaxis (urticaria, decreased blood pressure, and dyspnea) have occurred with RADICAVA.

Patients should be monitored carefully for hypersensitivity reactions. If hypersensitivity reactions occur, discontinue RADICAVA or RADICAVA ORS, treat per standard of care, and monitor until the condition resolves.

Sulfite Allergic Reactions
RADICAVA and RADICAVA ORS contain sodium bisulfite, a sulfite that may cause allergic-type reactions, including anaphylactic symptoms and life-threatening or less severe asthmatic episodes in susceptible people. The overall prevalence of sulfite sensitivity in the general population is unknown but occurs more frequently in asthmatic people.

Adverse Reactions
The most common adverse reactions (≥10%) reported in RADICAVA-treated patients were contusion (15%), gait disturbance (13%), and headache (10%). In an open label study, fatigue was also observed in 7.6% of patients receiving RADICAVA ORS.

Pregnancy
Based on animal data, RADICAVA and RADICAVA ORS may cause fetal harm.

To report suspected adverse reactions or product complaints, contact Mitsubishi Tanabe Pharma America, Inc., at 1-888-292-0058. You may also report suspected adverse reactions to the FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.

INDICATION
RADICAVA and RADICAVA ORS are indicated for the treatment of amyotrophic lateral sclerosis (ALS).

For more information, including full Prescribing Information, please visit www.RADICAVA.com.

About Mitsubishi Tanabe Pharma America, Inc.
Based in Jersey City, N.J., Mitsubishi Tanabe Pharma America, Inc. (MTPA) is a wholly-owned subsidiary of Mitsubishi Tanabe Pharma Corporation (MTPC). It was established by MTPC to develop and advance our pipeline as well as commercialize approved pharmaceutical products in North America. For more information, please visit www.mt-pharma-america.com or follow us on X (formerly Twitter), Facebook and LinkedIn.

About Mitsubishi Tanabe Pharma Corporation
Mitsubishi Tanabe Pharma Corporation (MTPC), the pharma arm of Mitsubishi Chemical Group (MCG), is one of the oldest pharmaceutical companies in the world, founded in 1678. MTPC is headquartered in Doshomachi, Osaka, the birthplace of Japan's pharmaceutical industry. MCG has positioned health care as its strategic focus in its management policy, "Forging the future". MTPC sets the MISSION of "Creating hope for all facing illness". To that end, MTPC is working on the disease areas of central nervous system, immuno-inflammation, diabetes and kidney, and cancer. MTPC is focusing on "precision medicine" to provide drugs with high treatment satisfaction and additionally working to develop "around the pill solutions" to address specific patient concerns based on therapeutic medicine, including prevention of diseases, pre-symptomatic disease care, prevention of aggravation and prognosis. For more information, go to https://www.mt-pharma.co.jp/e/.

Media inquiries:
Media_MTPA@mt-pharma-us.com 

¹ RADICAVA and RADICAVA ORS Prescribing Information. Jersey City, NJ: Mitsubishi Tanabe Pharma America, Inc.; 2022.
² Shimizu H, et al. Bioequivalence study of oral suspension and intravenous formulation of edaravone in healthy adult subjects. Clin Pharmacol Drug Dev. 2021;10(10):1188-1197.
³ Data on file. Mitsubishi Tanabe Pharma America, Inc.
⁴ Data on file. Mitsubishi Tanabe Pharma America, Inc.
⁵ Data on file. Mitsubishi Tanabe Pharma America, Inc.

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SOURCE Mitsubishi Tanabe Pharma America