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

By BioMarin Pharmaceutical Inc., 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

BioMarin's planned submission to global health authorities on track for second half of 2025

SAN RAFAEL, Calif. , Sept. 6, 2025 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced new data characterizing the efficacy and safety of PALYNZIQ ® (pegvaliase-pqpz) for the treatment of adolescents with phenylketonuria (PKU), which were presented at the 15th International Congress of Inborn Errors of Metabolism (ICIEM) in Kyoto, Japan , Sept. 2-6, 2025 .

The Phase 3 PEGASUS study evaluating the efficacy and safety of PALYNZIQ in