Mitsubishi Tanabe Pharma America Unveils New RADICAVA® (edaravone) Results for ALS at ENCALS 2024

Mitsubishi Tanabe Pharma America, Inc. (MTPA) recently released findings from two significant studies on RADICAVA® (edaravone) for the treatment of amyotrophic lateral sclerosis (ALS). These results were presented at the European Network to Cure Amyotrophic Lateral Sclerosis (ENCALS) 2024 meeting. The studies suggest a notable survival benefit and reinforce the drug's safety profile over extended use.

The first study utilized data from the ALS/MND Natural History Study Consortium. This ongoing real-world analysis compared 176 individuals treated with RADICAVA in combination with riluzole to another 176 individuals treated with riluzole alone. The findings indicate a survival benefit of 3.2 additional months for those receiving RADICAVA alongside riluzole, compared to those on riluzole alone. The survival time for the RADICAVA group was 31.1 months versus 28.8 months for the riluzole-only group, showcasing a statistically significant improvement.

Dr. Gustavo A. Suarez Zambrano, Vice President of Medical Affairs at MTPA, emphasized the importance of real-world data in understanding and treating complex diseases like ALS. He expressed pride in collaborating with the ALS/MND Consortium, highlighting the significance of these real-world insights for the medical community.

The second study, labeled MT-1186-A03, was a Phase 3, open-label, multi-center extension study that evaluated the long-term safety of RADICAVA ORS® over a 96-week period. This study built upon the initial 48-week trial (MT-1186-A01) and continued to affirm the safety and tolerability of the drug. Participants received a 105-mg dose of RADICAVA ORS according to FDA-approved guidelines. Common adverse events reported included falls, muscular weakness, dyspnea, constipation, and dysphagia, consistent with previous safety data.

Alex Sherman, Director of the Center for Innovation and Biomedical Informatics (CIB) at the NCRI at MGH and Principal Investigator for the study, highlighted the collaborative effort with MTPA. He noted that the real-world findings bolster confidence in existing treatment options and underscore a commitment to improving patient outcomes.

RADICAVA (edaravone) was first approved by the U.S. Food and Drug Administration (FDA) in 2017, with the oral formulation, RADICAVA ORS, receiving approval in 2022. This medication is designed for use in 28-day cycles, administered via intravenous infusion or taken orally, depending on the formulation. RADICAVA is administered in 14-day cycles initially, followed by 10-day cycles in subsequent treatments, with each cycle including a drug-free period.

The drug was discovered and developed by Mitsubishi Tanabe Pharma Corporation (MTPC) and has received regulatory approval in multiple countries, including Japan, South Korea, Canada, Switzerland, and several others. It has been used to treat over 16,000 individuals with ALS in the United States alone.

Safety information for RADICAVA includes possible hypersensitivity reactions and sulfite allergic reactions. Common adverse effects reported in clinical trials include contusion, gait disturbance, headache, and fatigue.

Overall, the findings from these studies reinforce the added survival benefit and long-term safety of RADICAVA for ALS patients, highlighting its importance as a treatment option.