Long‐term safety of oral edaravone in Japanese patients with amyotrophic lateral sclerosis: Sub‐analysis of a global, open‐label, phase 3 study

AbstractIntroduction/AimsPatients with amyotrophic lateral sclerosis (ALS) experience a slower rate of physical function decline when treated with intravenous edaravone. The oral suspension formulation of edaravone (105 mg) has a similar pharmacokinetic profile to intravenous edaravone (60 mg/60 min). The long‐term safety of oral edaravone in Japanese patients with ALS has not been reported. Therefore, a sub‐analysis of Japanese patients in a global phase 3 study (NCT04165824) was conducted to evaluate the safety and tolerability of oral edaravone in Japanese patients with ALS.MethodsThis study was a global, open‐label, phase 3 study to evaluate the long‐term safety and tolerability of oral edaravone in patients with ALS. Patients with ALS received oral edaravone (105 mg/day) for 48 weeks. Adverse events in Japanese patients were assessed at week 48.ResultsAmong the 185 patients enrolled globally, 65 patients were enrolled in Japan (mean age, 59.3 years; mean disease duration, 1.5 years). Most patients experienced treatment‐emergent adverse events (TEAEs) (84.6%), and the most common TEAEs by week 48 were constipation (15.4%), insomnia (12.3%), and back pain (10.8%). Two serious TEAEs were reported: atrial fibrillation and pleural effusion (both n = 1). Three adverse drug reactions (ADRs) were reported: diarrhea, abnormal hepatic function, and fatigue (all n = 1). There were no serious ADRs or TEAEs/ADRs that led to study drug discontinuation.DiscussionOral edaravone had a similar safety profile to intravenous edaravone in Japanese patients, and good tolerability over 48 weeks. No new safety concerns were observed in this population.