Mitsubishi Tanabe Pharma America Unveils New RADICAVA ORS® Findings at NEALS 2024

In a recent announcement, Mitsubishi Tanabe Pharma America, Inc. (MTPA) revealed significant findings from two studies involving RADICAVA ORS® (edaravone) at the 2024 Annual Meeting of the Northeast Amyotrophic Lateral Sclerosis Consortium (NEALS). The results from the MT-1186-A04 Study, a Phase 3b extension of the MT-1186-A02 study, were shared alongside primary and post-hoc analyses comparing RADICAVA ORS with historical placebo controls from the Pooled Resource Open-Access ALS Clinical Trials (PRO-ACT). The data indicated that RADICAVA ORS might enhance survival rates and reduce the decline in physical function in ALS patients compared to the PRO-ACT placebo group.

Gustavo A. Suarez Zambrano, M.D., Vice President of Medical Affairs at MTPA, expressed the company's enthusiasm about the findings, emphasizing their potential value to the ALS and medical communities and reinforcing MTPA's commitment to providing impactful insights.

The MT-1186-A04 study was a randomized, double-blind, multi-center extension study, monitoring patients for an additional 48 weeks following the MT-1186-A02 study. In this extension, patients who had already been treated with either investigational once-daily dosing or the FDA-approved on/off regimen of RADICAVA ORS were followed. Results indicated that daily dosing was not superior to the on/off dosing in reducing ALS Functional Rating Scale-Revised (ALSFRS-R) scores, affirming that the on/off regimen remains the most suitable for ALS patients.

Additionally, MTPA presented analyses comparing the long-term function and survival of RADICAVA ORS-treated patients with matched historical controls from PRO-ACT. These analyses, involving propensity score-matched cohorts on various baseline variables, suggested that long-term treatment with RADICAVA ORS contributed to slower functional decline and improved survival outcomes in ALS patients compared to those in the PRO-ACT placebo group.

RADICAVA ORS received FDA approval on May 12, 2022, for the treatment of amyotrophic lateral sclerosis (ALS), following the earlier approval of the intravenous formulation, RADICAVA, on May 5, 2017. The FDA granted RADICAVA ORS Orphan Drug Exclusivity in 2024 due to its significant contribution to patient care through its innovative oral formulation. RADICAVA is administered in a 28-day cycle via intravenous infusion, whereas RADICAVA ORS is taken orally under a similar regimen but with specific guidelines on fasting and intake timing.

Edaravone, the active ingredient in RADICAVA and RADICAVA ORS, was developed by Mitsubishi Tanabe Pharma Corporation (MTPC) and has been commercialized in the U.S. by MTPA. MTPC has been conducting research on ALS since 2001, with edaravone first approved for ALS treatment in Japan and South Korea in 2015 under the brand RADICUT®. Subsequent approvals followed in several other countries, including Canada, Switzerland, Indonesia, Thailand, Malaysia, and Brazil, with regulatory nods for the oral suspension formulation in Canada, Switzerland, and Japan.

Since its introduction in the U.S., RADICAVA and RADICAVA ORS have been administered to over 16,000 ALS patients, accumulating over 2.0 million days of therapy and prescribed by more than 2,400 healthcare providers.

It is crucial to monitor for hypersensitivity and sulfite allergic reactions in patients receiving RADICAVA or RADICAVA ORS. The most frequent adverse reactions include contusion, gait disturbance, and headache. Additionally, fatigue was reported in some patients during an open-label study. For pregnant patients, animal data suggests potential fetal harm.

RADICAVA and RADICAVA ORS are specifically indicated for the treatment of amyotrophic lateral sclerosis (ALS).