BioMarin Reports Positive Phase III Results for Palynziq in Adolescent PKU Patients

BioMarin Pharmaceutical has announced that its Phase III PEGASUS trial of Palynziq (pegvaliase-pqpz) in adolescents with phenylketonuria (PKU), also known as PAH deficiency, met its primary efficacy endpoint. The trial demonstrated a significant reduction in blood phenylalanine (Phe) levels in patients aged 12–17 compared to those managed with dietary therapy alone. The randomized, open-label study involved 55 participants and assessed both efficacy and safety over a 73-week primary treatment phase, followed by an optional 80-week extension. Participants in the diet-only group were given the opportunity to cross over to treatment in the second phase. Secondary outcomes included changes in protein intake and pharmacokinetics, with safety findings consistent with previous data, primarily involving immune-mediated responses. Palynziq, a PEGylated version of phenylalanine ammonia lyase, substitutes the deficient PAH enzyme to metabolize Phe. BioMarin intends to submit the full trial results to regulatory authorities later this year, seeking label expansion to include adolescents.

03-04-2025

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