Efficacy and safety of pegzilarginase in arginase 1 deficiency (PEACE)

Pegzilarginase, a novel human arginase 1 enzyme therapy, shows promise as the first potential treatment for Arginase 1 Deficiency (ARG1-D). A rare inherited metabolic disease causing increased morbidity and premature mortality, ARG1-D lacks effective therapies. In a trial from May 2019 to March 2021 with 32 patients, pegzilarginase significantly reduced plasma arginine levels, normalizing them in 90.5% of patients compared to a placebo. The treatment also demonstrated clinically relevant improvements in functional mobility, sustained over 24 additional weeks. Well-tolerated with mostly mild adverse events, pegzilarginase emerges as a potential breakthrough for ARG1-D patients.