PEGASUS Study

BioMarin announced positive Phase 3 PEGASUS study results for PALYNZIQ® in adolescents (12-17) with PKU, presented at ICIEM 2025. PALYNZIQ demonstrated a 49.7% decrease in mean blood Phe levels compared to diet alone. A significant proportion of PALYNZIQ-treated participants achieved target Phe levels, with a safety profile consistent with adult patients. BioMarin plans to submit these data to global health authorities in the second half of 2025, seeking to expand PALYNZIQ’s indication to include adolescents.