pegzilarginase

Pegzilarginase is a pegylated recombinant human arginase 1 enzyme therapy developed initially by Aeglea BioTherapeutics and later acquired globally by Immedica for arginase 1 deficiency, a rare urea cycle disorder characterized by hyperargininemia. It is designed to metabolize excess arginine and thereby lower plasma arginine and related toxic metabolites, addressing the core metabolic defect that drives neurologic and functional impairment in ARG1-D. Clinical development included the pivotal Phase 3 PEACE study, which showed significant arginine lowering and supportive clinical benefit, and the product has been marketed as Loargys in Europe and Great Britain and approved in the United States as Loargys for hyperargininemia in adults and pediatric patients aged 2 years and older with arginase 1 deficiency, in conjunction with dietary protein restriction. It is administered as a recombinant enzyme product and represents a disease-modifying metabolic therapy rather than a symptomatic treatment.

Brand names

Loargys

Other names

pegzilarginase-nbln