Real-World Study Suggests Fewer ALS Progression Milestones With Edaravone

Treatment with intravenous (IV) edaravone was associated with fewer reported disease progression milestones and deaths in patients with amyotrophic lateral sclerosis (ALS), according to real-world data published in the Journal of Comparative Effectiveness Research.

ALS is a neurodegenerative condition that results in cell neuron death and progressive muscular weakness. The IV formulation of edaravone was approved under the brand name Radicava^® in May 2017 for the treatment of ALS.

The retrospective, observational, comparative effectiveness cohort study utilized health claims data from the Optum Clinformatic^® Data Mart, a database composed of both commercial and Medicare Advantage plan members. The study included data from people aged 18 years and older (on the date of the first claim) diagnosed with ALS who had previously been treated with IV edaravone between August 8, 2017 and September 31, 2021 and who may or may not have received treatment with riluzole.

Study participants with ALS and at least 1 claim for IV edaravone (n=395) were propensity score matched 1:1 with participants not treated with IV edaravone (control; n=395). Restricted mean time lost was assessed for the 6 disease progression milestones: use of gait assistive devices (canes/walkers/wheelchairs), artificial nutrition, noninvasive ventilation, invasive ventilation, speech-generating devices, and hospice. 

Findings showed IV edaravone-treated patients had longer disease progression milestone-free time for all 6 milestones compared with those not treated with IV edaravone. From 0 to 24 months, more patients treated with IV edaravone reported no milestones compared with those not treated. Additionally, fewer deaths were reported among treated patients vs controls.

“These data reinforce the utility of Radicava and build upon the growing evidence that expands our knowledge of edaravone in ALS,” said Gustavo A. Suarez Zambrano, MD, Vice President of Medical Affairs at MTPA. “By continuing to study Radicava in both its IV and oral suspension forms, we aim to expand our understanding of the medication and its limitations to better support the ALS community.”

The Company did note that given the study’s design, results may not be generalizable and should not be used to determine definitive conclusions about treatment effects.

This article originally appeared on MPR