50% Blood Phe Reduction: BioMarin's PALYNZIQ Breakthrough for Teenage PKU Patients Outperforms Diet Therapy

BioMarin Announces Positive Pivotal Phase 3 Data for PALYNZIQ® (pegvaliase-pqpz) in Adolescents with Phenylketonuria at 15th International Congress of Inborn Errors of Metabolism

New data from Phase 3 PEGASUS study demonstrates a 49.7% decrease in mean blood Phe levels in adolescents aged 12-17 treated with PALYNZIQ

50% Blood Phe Reduction: BioMarin's PALYNZIQ Breakthrough for Teenage PKU Patients Outperforms Diet Therapy

Phase 3 PEGASUS trial shows PALYNZIQ reduced blood Phe by 49.7% in adolescents with PKU. 51.6% achieved target Phe levels ≤600 µmol/L. Filing for expanded indication planned for H2 2025.