JERSEY CITY, N.J., December 10, 2021 – Mitsubishi Tanabe Pharma America, Inc. (MTPA) today announced virtual poster presentations reviewing the Phase 4 REFINE-ALS biomarker study in people with amyotrophic lateral sclerosis (ALS), including an overview of biomarker assays utilized in the study as well as COVID-19 mitigation strategies deployed through protocol amendments. Details were presented as part of the Motor Neurone Disease Association (MNDA) 32nd International Symposium on ALS/MND, being held virtually December 7-10.
“ALS patient needs remain MTPA’s primary focus, so it was a priority to address the evolving concerns of the ALS community during the pandemic and offer appropriate alternatives for patients to participate in the REFINE-ALS study through a virtual setting,” said Atsushi Fujimoto, President, MTPA. “The collective goal has always remained twofold – ensuring the safety of the participants while continuing to foster scientific research, including the use of optimized biomarker assays to obtain real-world data and help better understand this progressive disease.”
The REFINE-ALS study, conducted in collaboration with the Massachusetts General Hospital (MGH) Neurological Clinical Research Institute (NCRI), is designed to identify and measure specific biomarkers and clinical assessments in up to 300 people with ALS in the U.S. who have begun treatment on RADICAVA® (edaravone). The study is utilizing the expertise of multiple specialty laboratories to assess biomarker samples for oxidative stress, inflammation, neuronal and muscle injury. Biomarkers are measured prior to initiating treatment with RADICAVA, at the start of treatment, and at pre-specified time points throughout the 24-week study period (six cycles of treatment).
Protocol amendments to the study were implemented in January 2021, giving patients the ability to consent to participate virtually, with the option to utilize telemedicine and home health agencies instead of being required to go to the hospital or in-office visits. Trained nurses are responsible for collecting blood and urine samples during study visits to the clinic or at the participant’s home, with these samples being processed and shipped for same-day delivery to a central collection company.
“Researchers have faced many challenges over the last year, but our commitment to learning more about ALS and response to treatment with RADICAVA in a real-world setting has remained constant,” said primary investigator James Berry, M.D., M.P.H., MGH NCRI, Boston. “Our ongoing, adaptable collaboration with MTPA has allowed the REFINE-ALS study to continue enrollment and analyses of these complex biomarkers as a way to enhance understanding of ALS treatment.”
As of April 2021, over 30 sites are actively participating across North America. Patients interested in learning more about the REFINE-ALS study can call (617) 724-2609 or visit ClinicalTrials.gov (NCT04259255) for more information.
For more information on the REFINE-ALS poster presentations, click here.
About RADICAVA® (edaravone)
The U.S. Food and Drug Administration (FDA) approved RADICAVA® (edaravone) IV on May 5, 2017 as a treatment for amyotrophic lateral sclerosis (ALS).1 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).
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.
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 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/.
- RADICAVA® (edaravone) U.S. Prescribing Information. March 2021.