Positive pivotal data for Palynziq in adolescents

3 April 2025

US biotech BioMarin Pharmaceutical (Nasdaq: BMRN) yesterday revealed that the Phase III PEGASUS trial evaluating Palynziq (pegvaliase-pqpz) met its primary efficacy endpoint, demonstrating a statistically-significant lowering in blood phenylalanine( Phe) levels in adolescents aged 12-17 with phenylketonuria (PKU) compared to diet alone.

Safety results were consistent with the known profile of the medicine. Palynziq is the first and only enzyme therapy approved to treat adults with PKU, said BioMarin, whose shares edged up 3% to $68.87 following the announcement.

Detailed results from the PEGASUS study will be presented at an upcoming medical meeting and submitted to global health authorities starting later this year to request a label expansion for Palynziq to include adolescents.

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