Edaravone Treatment for Amyotrophic Lateral Sclerosis Real-World Study Results Similar to Pivotal Trials 

In a postapproval cohort study, individuals with amyotrophic lateral sclerosis (ALS) treated with edaravone (Radicava; Mitsubishi Tanabe Pharma, Jersey City, NJ) had a mean reduction of 12.9 points on the ALS Functional Rating Scale-Revised (ALSFRS-R). On average, participants had a monthly reduction of 0.40 +/- 0.52 points, going from 40.5 +/- 7.2, of a maximum 48 points possible, at the start of treatment; 27.6 +/- 11.8 at the last treatment visit .

This rate of change was consistent with that observed in a pivotal phase 3 trial (Study 19) (NCT04259255) that showed treatment with edaravone slowed the loss of physical function over a 24-week period by 33% or 2.49 ALSFRS-R points (P=.0013) compared with placebo. These results were presented at the Motor Neurone Disease Association (MNDA) 31st International Symposium on ALS/MND held virtually on December 9 to 11, 2020.

"We are pleased to share this real-world data with the ALS community," said Atsushi Fujimoto, president, MTPA. "We hope the insights are helpful for the healthcare providers who are considering treatment for those impacted by this debilitating disease."
By the end of the study, 78 participants (46.7%) who continued treatment and had repeat ALSFRS-R scores, 38.3% had more than 12 months of continuous treatment, and a median treatment duration of 22.6 months. Of the 167 participants, 89 (53.5%) discontinued treatment. Of the participants, 25% who continued treatment for 12 months reported feeling good in their overall health and wellness and 66.7% reported feeling okay.
The observational retrospective cohort study included 167 participants with ALS and a mean age of 64 years. Participants included in this analysis were treated with IV edaravone for at least 3 continuous months between August 8, 2017 and March 31, 2020. The study was limited to ALS participants receiving treatment through infusion services, so results may not be generalizable to all individuals with ALS. The study relies on electronic health record (EHR) data and self-reported data.