JERSEY CITY, N.J., January 7, 2021 – Mitsubishi Tanabe Pharma America, Inc. (MTPA) today announced revisions to the REFINE-ALS biomarker study protocol to address the safety of people with amyotrophic lateral sclerosis (ALS) during the pandemic. The amendment gives patients the ability to consent to participate virtually, with the option to utilize telemedicine and home health agencies in lieu of hospital or in-office visits.
“In an effort to address the evolving concerns of the ALS community this year, we made it a priority to do what we can to support them during these unprecedented times,” said Gustavo A. Suarez Zambrano, M.D., Vice President of Medical Affairs, MTPA. “With that in mind, we are thankful to be able to offer a potential alternative for patients to be able to participate in the REFINE-ALS study in a virtual setting when in-person assessments are not possible, enabling us to continue fostering scientific knowledge.”
The study, sponsored by MTPA and led by 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). There are approximately 40 sites across the country and the study will utilize the expertise of multiple specialty laboratories to assess biomarker samples for oxidative stress, inflammation, neuronal and muscle injury. Biomarkers will be 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).
In addition, patients may opt in to receive their genetic results for five common mutations associated with the disease, including SOD1, TARDBP, C9ORF72, FUS and VCP. Advanced tele-genetic counseling will also be made available for those who choose to receive this information.
“There is much about ALS that we still don’t know, but we’re working toward learning more every day,” said primary investigator James Berry, M.D., M.P.H., MGH NCRI, Boston. “We are grateful to MTPA for their support with these important changes to the REFINE-ALS study, and excited to be able to share with participants more about their genetic makeup in relation to ALS. I generally see research as a way forward to better understand this progressive disease, and for many patients, participating in a study that evaluates biomarkers is a way of giving back to the ALS community at large.”
Patients interested in learning more about the trial can call (617) 724-2609 or visit ClinicalTrials.gov (NCT04259255) for more information.
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).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. 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 use as a treatment for ALS in Japan and South Korea. Marketing authorization was granted in Canada in October 2018 and Switzerland in January 2019.
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.
Overview of Mitsubishi Tanabe Pharma Corporation
Mitsubishi Tanabe Pharma, which was founded in 1678, has its headquarters in Doshomachi, Osaka, which is the birthplace of Japan’s pharmaceutical industry. With business centered on ethical pharmaceuticals, Mitsubishi Tanabe Pharma is a well-established company with one of the longest histories of pharmaceutical companies in Japan.2 In accordance with the corporate philosophy of “contributing to the healthier lives of people around the world through the creation of pharmaceuticals,” the Company formulated the key concept of Open Up the Future under the Medium-Term Management Plan 2016-2020. Through the discovery of drugs that address unmet medical needs, centered on its priority disease areas — immune-inflammation diseases, diabetes and kidney, central nervous system, and vaccines — Mitsubishi Tanabe Pharma will strive to contribute to the health of patients around the world. MTPC is the parent company of MTPA and MTDA. For more information, go to https://www.mt-pharma.co.jp/e/.
- RADICAVA® U.S. Prescribing Information. August 2018.
- Research by TOKYO SHOKO RESEARCH, LTD.