Mechanism of action and clinical trial results of a new drug for amyotrophic lateral sclerosis (ALS), Mecobalamin (Rozebalamin®) for intramuscular injection, 25 mg

Key Points

• To investigate the mechanism of action and clinical efficacy of mecobalamin in treating amyotrophic lateral sclerosis (ALS).
• Conducted a Phase III double-blind placebo-controlled trial focusing on ALS patients within one year of diagnosis.
• Evaluated the primary outcome using the revised ALS Functional Rating Scale.
• Monitored patients for safety and tolerability of mecobalamin administration.
• The high-dose mecobalamin group showed a significant reduction in the decline of the ALS Functional Rating Scale scores.
• Safety assessments confirmed the acceptability of mecobalamin, with no severe adverse events reported.