No unexpected safety signals were reported in the post hoc analysis.
Patients with bulbur-onset amyotrophic lateral sclerosis (ALS) who receive treatment with edaravone demonstrate a reduction in the ALS Functional Rating Score, Revised (ALSFRS-R) regardless of forced vital capacity (FVC), according to findings from a post hoc analysis.1
The results were presented during the virtual Clinical Trials Session of the 2020 Muscular Dystrophy Association (MDA) Clinical and Scientific Conference on March 24, 2020.
In the study, patients with bulbar-onset ALS with either FVC >80% or <80% experienced a reduction in ALSFRS-R score loss compared with the placebo group.
Edaravone was approved by the FDA in May 2017 for the treatment of ALS. Administered as an intravenous (IV) infusion, it is marketed under the brand name Radicava.
The approval of IV edaravone was based off results from the completed Study 19 (NCT01492686). Study 19 included 137 patients with ALS who were randomized to receive either 60 mg edaravone or placebo over a 24-week treatment period. In that study, researchers documented a least square mean change of -5.01 (standard error [SE], 0.64) from baseline for ALSFRS-R scores with the neuroprotective drug compared with a change of -7.50 (SE, 0.66) for placebo. That 2.49-point difference (SE, 0.76; 95% CI, 0.99—3.98; P = .0013) represented a 33% improvement in ALSFRS-R in patients with reduced FVC of <80% prior to starting edaravone versus placebo.2
In this post hoc analysis, researchers aimed to evaluate the efficacy of edaravone in patients with bulbur-onset ALS and specifically those with FVC of either >80% or <80%.1
Through 48 weeks, patients in both the bulbar and limb-onset groups experienced a reduction in ALSFRS-R score loss versus placebo following treatment. Patients with bulbur-onset ALS with either >80% or <80% experienced similar ALSFRS-R scores, indicating the drug’s efficacy regardless of FVC.1
Other observations in the study included a notable change in the slope of the ALSFRS-R score-vs-time graph from baseline to week 48 in former placebo patients with either bulbar or limb-onset ALS. Additionally, there were no unexpected safety signals, nor any inconsistencies with the results from Study 19.1
1. Pattee G, Suarez Zambrano G, Zhang J, Nelson S, Apple S. Post hoc analysis of edaravone study 19: efficacy in bulbar onset ALS patients with and without reduced pulmonary function. Presented at: 2020 MDA Clinical & Scientific Conference. Abstract 53.
2. Safety and efficacy of edaravone in well defined patients with amyotrophic lateral sclerosis: a randomised, double-blind, placebo-controlled trial. Lancet Neurol. 2017;16(7):505-512. doi: 10.1016/S1474-4422(17)30115-1