US FDA approves Mitsubishi Tanabe Pharma

Mitsubishi Tanabe Pharma America, Inc. (MTPA) announced the US Food and Drug Administration (FDA) has approved Radicava ORS (edaravone), the oral form of edaravone, for the treatment of amyotrophic lateral sclerosis (ALS), a neurodegenerative disease that currently has no cure and can progress rapidly. Radicava ORS offers the same efficacy as Radicava (edaravone), an FDA-approved intravenous (IV) treatment shown in a pivotal trial to help slow the loss of physical function in ALS. "At MTPA, patients have been the driving force behind our work as we strive to develop meaningful treatment options for ALS and continue to tackle unmet needs," said Atsushi Fujimoto, President, MTPA. "Five years ago, we proudly launched Radicava as a treatment option for patients with ALS in the US Now, we continue to push the boundaries of innovation with Radicava ORS, an orally administered option allowing patients flexibility in how they take their medicine." Radicava ORS is specifically formulated for patients with ALS and provides a flexible administration option (taken orally or via feeding tube) with a small, 5 mL dose, a portable bottle, 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.1 RADICAVA ORS should be taken in the morning after overnight fasting. "ALS is a progressive disease that, due to its heterogenous nature, impacts patients at different rates with varying symptoms," said Tulio Bertorini, M.D., Professor of Neurology, The University of Tennessee Health Science Center. "Therefore, it is crucial that patients have treatment and formulation options that accommodate their own unique needs, and Radicava ORS provides HCPs who have prescribed their ALS patients edaravone with an alternate delivery option." The comprehensive clinical development program for edaravone in ALS has spanned over a decade and included multiple clinical trials for the IV and oral formulations. The FDA approval of Radicava ORS is supported by several studies, including data from the pivotal phase 3 clinical trial (MCI186-19) evaluating 137 ALS patients that showed treatment with Radicava slowed the loss of physical function by 33 per cent (approximately one-third) compared to placebo at 24 weeks, as measured by the ALS Functional Rating Scale-Revised (ALSFRS-R), a validated rating instrument for monitoring disease progression in patients. Additionally, seven Phase 1 clinical pharmacology studies were conducted to examine the pharmacokinetics, safety, drug-to-drug interactions, dosing, bioavailability and bioequivalence of Radicava ORS in healthy individuals and ALS patients with and without a percutaneous endoscopic gastrostomy (PEG) tube/nasogastric (NG) tube, as well as a global Phase 3, 24-week trial demonstrating the safety and tolerability profile of the treatment in 185 patients with ALS. Lastly, there is an ongoing phase 3 study evaluating the long-term safety and tolerability of Radicava ORS for up to 96 weeks. The most common adverse events that occurred in greater than 10 per cent of patients treated with Radicava were bruising (contusion), problems walking (gait disturbance) and headache.1 Fatigue was observed in 7.6 per cent of patients taking Radicava ORS. Radicava and Radicava ORS are contraindicated in patients with a history of hypersensitivity to edaravone or any of the inactive ingredients of this product. Hypersensitivity reactions and anaphylactic reactions have been reported in patients treated with Radicava. "To address the unmet needs in ALS, we must continue to find new treatments, as well as optimize current treatments and care," said Calaneet Balas, president & CEO of The ALS Association. "We thank MTPA and the FDA for working diligently to make this oral formulation possible and are confident it will be a welcome addition to help further support the ALS community." As a part of MTPA's commitment to helping patients with ALS access Radicava and Radicava ORS, MTPA created the JourneyMate Support Program, which provides resources to help patients and caregivers on their ALS treatment journey. The JourneyMate Support Program offers educational support and resources for patients who are considering or have already been prescribed an MTPA product. Experienced program team members are trained to address patient and caregiver educational needs and provide them with personalized answers and resources for living with ALS. ALS Resource Specialist: A go-to resource in the JourneyMate Support Program for general information about ALS and Radicava ORS. Insurance & Access Specialist: This specialist can help patients and caregivers understand insurance coverage, financial support options, site of care, specialty pharmacy options and the steps to accessing a Radicava ORS prescription. ALS Clinical Educator: Once Radicava ORS is prescribed, an ALS Clinical Educator can provide personalized education to patients and their families about Radicava ORS and will also provide resources throughout treatment. The US 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). Radicava is administered in 28-day cycles by 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. Edaravone was discovered and developed for ALS by Mitsubishi Tanabe Pharma Corporation (MTPC) and Mitsubishi Tanabe Pharma Development America, Inc (MTDA), commercialized in the US 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, Radicava 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), Thailand (April 2021) and Malaysia (December 2021). To date, in the US, Radicava has been used to treat over 6,500 patients, with nearly one-million days of therapy, and has been prescribed by more than 1,600 HCPs. Mitsubishi Tanabe Pharma America, Inc. (MTPA) is a wholly-owned subsidiary of Mitsubishi Tanabe Pharma Corporation's (MTPC) 100 per cent owned US holding company, Mitsubishi Tanabe Pharma Holdings America, Inc.

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