Mitsubishi Tanabe Pharma America Announces FDA Acceptance of New Drug Application (NDA) for Oral Edaravone Formulation for the Treatment of ALS

JERSEY CITY, N.J., Jan. 12, 2022 /PRNewswire/ -- Mitsubishi Tanabe Pharma America, Inc. (MTPA) today announced the U.S. Food and Drug Administration (FDA) has accepted the new drug application (NDA) for an investigational oral formulation of edaravone (MT-1186) for the treatment of amyotrophic lateral sclerosis (ALS). The FDA has accepted the application for priority review with a Prescription Drug User Fee Act (PDUFA) action date of May 12, 2022. The FDA grants Priority Review for medicines that, if approved, would be significant improvements in the safety or effectiveness of the treatment, diagnosis, or prevention of serious conditions when compared to standard applications.¹

Investigational oral edaravone has been developed to have a similar clinical profile compared to the intravenous (IV) formulation of RADICAVA^® (edaravone), which was approved by the FDA for the treatment of ALS in May 2017.²

"At MTPA, we thrive on tackling the toughest challenges and are focused on addressing the unmet needs of the ALS community, which is illustrated by our ongoing development program for edaravone," said Atsushi Fujimoto, President, MTPA. "Our top priority is to help ensure ALS patients have flexibility with treatment and formulation options that are right for their specific needs. We look forward to working with the FDA in the coming months to bring this new formulation to patients as soon as possible."

The comprehensive clinical development program for edaravone in ALS has spanned over a decade and included multiple clinical trials for the IV and the investigational oral formulation. The NDA is supported by data from the RADICAVA IV pivotal trial and upwards of seven Phase 1 clinical pharmacology studies examining the pharmacokinetics, safety, drug-drug interactions, dosing, bioavailability and bioequivalence of the oral suspension in healthy individuals and ALS patients with and without PEG/NGT, along with a Phase 3 clinical trial evaluating the safety and tolerability of the investigational oral suspension over the course of 24 weeks in patients with ALS. There is an ongoing study evaluating the safety and tolerability of the oral suspension at 48 and 96 weeks.

"Oral edaravone may provide another formulation option for ALS patients looking for an alternate mode of administration," said Terry Heiman-Patterson, M.D., Professor, Neurology, Lewis Katz School of Medicine at Temple University, Director, Temple MDA/ALS Center of Hope. "We know that every patient living with ALS has their own unique needs, and I'm hopeful that an oral suspension formulation will offer a new and much needed option."

About Oral Edaravone (MT-1186)
Oral edaravone is an investigational oral formulation of edaravone being developed by Mitsubishi Tanabe Pharma Corporation (MTPC) and Mitsubishi Tanabe Pharma Development America, Inc (MTDA).

In 2019, Mitsubishi Tanabe Pharma group companies completed a series of Phase 1 studies comparing the oral formulation and intravenous formulations of edaravone and have started a global, multi-center, open-label, Phase 3 clinical trial evaluating safety and tolerability of oral edaravone in 185 patients with ALS across approximately 50 sites in the U.S., Canada, Europe and Japan. More information on the clinical trials is available at ClinicalTrials.gov (NCT04165824 and NCT04577404).

About RADICAVA^® (edaravone) IV
The U.S. Food and Drug Administration (FDA) approved RADICAVA^® (edaravone) IV on May 5, 2017 as a treatment for amyotrophic lateral sclerosis (ALS).² Edaravone was discovered and developed for ALS by Mitsubishi Tanabe Pharma Corporation (MTPC) and Mitsubishi Tanabe Pharma Development America, Inc (MTDA), commercialized in the U.S. by Mitsubishi Tanabe Pharma America, Inc. The MTPC group companies began researching ALS in 2001 through an iterative clinical platform over a 13-year period. In 2015, edaravone IV was approved for the treatment of ALS in Japan and South Korea. Marketing authorizations were subsequently granted in Canada (October 2018), Switzerland (January 2019), China (July 2019), Indonesia (July 2020), and Thailand (April 2021).

INDICATION
RADICAVA is indicated for the treatment of amyotrophic lateral sclerosis (ALS).

IMPORTANT SAFETY INFORMATION
Before you receive RADICAVA, tell your healthcare provider about all of your medical conditions, including if you:

• have asthma.
• are allergic to other medicines.
• are pregnant or plan to become pregnant. It is not known if RADICAVA will harm your unborn baby.
• are breastfeeding or plan to breastfeed. It is not known if RADICAVA passes into your breast milk. You and your healthcare provider should decide if you will receive RADICAVA or breastfeed.

Tell your healthcare provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements.

What are the possible side effects of RADICAVA?

• RADICAVA may cause serious side effects including hypersensitivity (allergic) reactions and sulfite allergic reactions.
• Hypersensitivity reactions have happened in people receiving RADICAVA and can happen after your infusion is finished.
• RADICAVA contains sodium bisulfite, a sulfite that may cause a type of allergic reaction that can be serious and life-threatening. Sodium bisulfite can also cause less severe asthma episodes in certain people. Sulfite sensitivity can happen more often in people who have asthma than in people who do not have asthma.
• Tell your healthcare provider right away or go to the nearest emergency room if you have any of the following symptoms: hives; swelling of the lips, tongue, or face; fainting; breathing problems; wheezing; trouble swallowing; dizziness; itching; or an asthma attack (in people with asthma).
• Your healthcare provider will monitor you during treatment to watch for signs and symptoms of all the serious side effects.

The most common side effects of RADICAVA include bruising (contusion), problems walking (gait disturbance), and headache.

These are not all the possible side effects of RADICAVA. Call your healthcare provider for medical advice about side effects. You may report side effects to Mitsubishi Tanabe Pharma America, Inc. at 1-888-292-0058 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.

For more information, including full Prescribing Information and Patient 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's (MTPC) 100 percent owned U.S. holding company, Mitsubishi Tanabe Pharma Holdings America, Inc. It was established by MTPC to commercialize approved pharmaceutical products in North America. For more information, please visit www.mt-pharma-america.com or follow us on Twitter, Facebook and LinkedIn.

About Mitsubishi Tanabe Pharma Development America, Inc.
The U.S. headquarters of Mitsubishi Tanabe Pharma Development America, Inc. (MTDA) is located in Jersey City, New Jersey. MTDA is a wholly-owned subsidiary of Mitsubishi Tanabe Pharma Corporation's 100 percent-owned U.S. holding company, Mitsubishi Tanabe Pharma Holdings America, Inc. For more information, please visit https://mt-pharma-development-america.com/

About Mitsubishi Tanabe Pharma Corporation
Mitsubishi Tanabe Pharma Corporation (MTPC), founded in 1678, is one of the oldest pharmaceutical companies in the world. Focused on ethical pharmaceuticals, MTPC is headquartered in Doshomachi, Osaka, the birthplace of Japan's pharmaceutical industry. In light of the anticipated changes in healthcare needs in the future, MTPC is advancing business activities under the mission of "Creating hope for all facing illness," and has set the corporate vision for 2030 (VISION 30) to "Be a healthcare company that delivers optimal therapy to each individual." As part of the vision for 2030, MTPC is prioritizing work on "precision medicine" to create effective therapies and preventive methods by identifying patient populations with high potential for efficacy and safety, focusing on the disease areas of central nervous system and immuno-inflammation. In addition, MTPC is 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. MTPC is a member of Mitsubishi Chemical Holdings Group. For more information, go to https://www.mt-pharma.co.jp/e/.

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¹ U.S. Food and Drug Administration. Priority Review. FDA website. Accessed November 2021. https://www.fda.gov/patients/fast-track-breakthrough-therapy-accelerated-approval-priority-review/priority-review.
² RADICAVA® (edaravone) U.S. Prescribing Information. March 2021.

SOURCE Mitsubishi Tanabe Pharma America, Inc.