Oral Edaravone Well-Tolerated as ALS Therapy

Oral edaravone (Radicava ORS; Mitsubishi Tanabe Pharma America [MTPA], Jersey City, NJ) was well-tolerated over 48 weeks in patients being treated for amyotrophic lateral sclerosis (ALS), according to updates from a phase 3 trial published in a recent issue of Muscle & Nerve. By week 48, the most common treatment-emergent adverse events (TEAEs) related to the medication were fatigue (n=6), dizziness (n=5), headache (n=4), and constipation (n=4). Overall, 24.9% of participants reported TEAEs related to edaravone, similar to reported TEAEs with the intravenous (IV) form of edaravone. No serious TEAEs related to the medication were reported by week 48, but 8.6% (n=16) of participants discontinued edaravone due to TEAEs.

MT-1186-A01 (NCT04165824) was a global, multicenter, open-label, phase 3 study over 48 weeks from November 2019 to October 2020 in 50 healthcare centers in 6 countries. Participants (n=185) received a 105-mg dose of edaravone in cycles to replicate the 4-week dosing schedule of IV edaravone. Cycles included 10 to 14 days of daily edaravone administration followed by 14 days of being drug-free. The primary objective was to monitor the safety and tolerability of oral edaravone over 48 weeks for the treatment of ALS. Safety was assessed at weeks 24 and 48. Participants were included if they were 18 to 75 years old; had definite, probable, or possible ALS; a baseline forced vital capacity (FVC) ≥ 70% of predicted; had ALS for at most 3 years; and could function independently. People were excluded if they had a malignant or pending biopsy, any other clinically significant disease process, alanine aminotransferase or aspartate aminotransferase elevations over 2 times the upper limit of normal, history of hypersensitivity to any of the ingredients in the medication, and inability to take oral medications.  Edaravone is contraindicated in people hypersensitive to any of the active ingredients in edaravone.

According to Gustavo A. Suarez Zambrano, MD, Vice President of Medical Affairs at MTPA, "These data build upon the study's 24-week findings that supported the FDA approval of Radicava ORS and underscore our commitment to growing our body of knowledge regarding the use of this treatment in people with ALS."

Radicava ORS (edaravone) was approved by the Food and Drug Administration as an oral suspension for the treatment of ALS on May 12, 2022. The IV formulation (Radicava) was approved as a treatment for ALS in May 2017. ALS is a progressive, neurodegenerative disease that causes systemic muscle weakness with approximately 5000 new cases of ALS diagnosed every year in the United States.