Short-Term Analysis Confirms Safety of Oral Edaravone in ALS Over 96 Weeks

Angela Genge, MD, FRCPC, eMBA

(Credit: McGill UniversityMcGill University)

Mitsubishi Tanabe Pharma America’s (MTPA) oral formulation of edaravone (Radicava ORS) demonstrated a consistent safety profile over 96 weeks in patients with amyotrophic lateral sclerosis (ALS), according to findings from the phase 3 MT-1186-A03 study (NCT04577404).¹ Presented at the 2025 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference, held March 16-19 in Dallas, Texas, these findings provided additional safety and tolerability reassurance for clinicians and patients considering long-term MTPA oral edaravone treatment in ALS.

The open-label, multi-center extension study evaluated the long-term safety of MTPA oral edaravone in patients who had completed the initial 48-week trial (MT-1186-A01). Over the additional 96-week period, the treatment remained well tolerated, and no new safety concerns were identified. The most common treatment-emergent adverse events (TEAEs) included fall, muscular weakness, dyspnea, constipation, and dysphagia—consistent with previously reported safety data for edaravone.

Presented by lead author Angela Genge, MD, FRCPC, eMBA, director of the ALS Clinic at The Neuro at Montreal Neurological Institute-Hospital, the MT-1186-A03 study followed participants who had completed 48 weeks of treatment in the preceding trial, MT-1186-A01 (NCT04165824). Eligible patients had a confirmed ALS diagnosis, a baseline forced vital capacity of at least 70%, and a disease duration of 3 years or less at study entry. All participants received MTPA oral edaravone at the FDA-approved 105-mg dose, following the same regimen established for the intravenous formulation.

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Building on clinical trial evidence supporting the safety and tolerability of oral edaravone, an additional real-world analysis of the ALS/Motor Neuron Disease (MND) Natural History Consortium (NHC) database suggested that edaravone treatment may also provide a survival benefit for patients with ALS. Also presented at the 2025 MDA Conference, the study compared patients with ALS receiving MTPA edaravone—either alone or alongside riluzole—to those treated with riluzole only.²

The ALS/MND NHC database, a clinic-based registry tracking longitudinal data on patients with ALS, was used to assess survival, treatment patterns, and clinical outcomes among those initiating MTPA edaravone. In the analysis, 169 patients receiving edaravone (± riluzole) were matched 1:1 to 169 patients on riluzole alone, balancing key demographic and clinical characteristics such as sex, age, BMI, disease duration, and ALS Functional Rating Scale-Revised scores.

After adjusting for baseline covariates, restricted mean survival time analysis suggested a survival benefit of 29.3 months for patients treated with edaravone versus 26.5 months for those receiving only riluzole, yielding a 3.2-month advantage (P <.03). Presented by lead author Alexander Sherman, MS, director of the Clinical Bioinformatics Neurological Clinical Research Institute at Massachusetts General Hospital, these findings offer valuable real-world insights into the potential benefits of edaravone beyond clinical trial settings.

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REFERENCES
1. Genge A, Pattee G, Sobue G, et al. Phase 3, Open-Label, Safety Extension Study of Radicava ORS® (Oral Edaravone) Administered Over 96 Weeks in Patients with ALS (MT-1186-A03). Presented at: 2025 MDA Clinical & Scientific Conference; March 16-19. Dallas, TX. Abstract P270.
2. Sherman A, Zhang J, Liu Y, et al. Survival Benefits and Treatment Patterns of Edaravone–Treated People With Amyotrophic Lateral Sclerosis in the ALS/MND Natural History Consortium. Presented at: 2025 MDA Clinical & Scientific Conference; March 16-19. Dallas, TX. Abstract P293.