Oral Edaravone Safe in Amyotrophic Lateral Sclerosis Through 144 Weeks

Among patients with amyotrophic lateral sclerosis (ALS), oral edaravone is found to be safe through 144 weeks with no new safety signals identified, according to study results presented at the 2025 American Academy of Neurology (AAN) annual meeting, held from April 5 to 9, 2025, in San Diego, California.

Intravenous (IV) edaravone, marketed as Radicava, was approved by the United States (US) Food and Drug Administration (FDA) for the treatment of ALS in 2017. In 2022, oral edaravone was approved by the US FDA after it was shown to have similar pharmacokinetic properties as the IV formulation.

The study MT-1186-A01 demonstrated that oral edaravone was well-tolerated through 48 weeks and was not associated with any new safety signals relative to IV edaravone.

To update the safety profile of oral edaravone with longer-term outcomes, researchers analyzed data from the extension study of MT-1186-A01 (ClinicalTrials.gov Identifier: NCT04165824) for the phase 3, open-label, multicenter trial, MT-1186-A03 (ClinicalTrials.gov Identifier: NCT04577404). The study population comprised patients with ALS who received 105 mg oral edaravone for 48 weeks in MT-1186-A01 and remained on oral edaravone for an additional 96 weeks.

At baseline, patients had a forced vital capacity of 70% or greater and had been diagnosed with definite, probable, probable-laboratory-supported, or possible ALS within the previous 3 years.

Oral edaravone was well-tolerated throughout the 96-week extension, or a total of 144 weeks, and no new safety signals were observed.

The most common treatment-emergent adverse events included fall, muscular weakness, dyspnea, constipation, and dysphagia.

The safety profile of oral edaravone was consistent with the known profile of edaravone observed in previous clinical trials.

“[Mitsubishi Tanabe Pharma America] oral edaravone showed no new safety concerns and was well-tolerated during the 96-week study period of MT-1186-A03, for a total of 144 weeks when including Study MT-1186.A01,” the researchers concluded.

Editor’s Note: A previous version of this article included the drug name “RadicavaÒ.” The correct product name is “Radicava.”

Disclosure: This research was supported by Mitsubishi Tanabe Pharma America Inc. Please see the original reference for a full list of disclosures