BioMarin Pharmaceutical(BMRN) Prnewswire·2025-04-02 13:00Core Insights - BioMarin Pharmaceutical Inc. announced that the Phase 3 PEGASUS trial for PALYNZIQ met its primary efficacy endpoint, showing a statistically significant reduction in blood Phe levels in adolescents aged 12-17 with phenylketonuria (PKU) compared to diet alone [1][2][3] - The company plans to submit detailed results to global health authorities later this year to request a label expansion for PALYNZIQ to include adolescents [2][3] Company Overview - BioMarin has been a pioneer in developing treatments for PKU for over two decades, with PALYNZIQ being the first and only enzyme therapy approved for adults with PKU [3][9] - The company aims to continue its innovation and research pipeline to provide meaningful options for patients transitioning to adult living [3] Study Details - The PEGASUS trial is a Phase 3 multi-center open-label randomized controlled study involving 55 adolescents aged 12-17 with PKU, focusing on the safety and efficacy of PALYNZIQ compared to diet alone [4][5] - The study consists of two parts: a primary treatment phase lasting 73 weeks and an extension phase of up to 80 weeks for monitoring [5] Product Information - PALYNZIQ works by substituting the deficient PAH enzyme in PKU with a PEGylated version to break down Phe, and it is administered with a dosing regimen designed for tolerability [6][12] - The product is currently approved for adults in the U.S. and for individuals aged 15 and older in Japan, among other regions, for those with uncontrolled blood Phe levels greater than 600 micromol/L [7][12] Patient Management - The management of PKU typically involves a strict Phe-restricted diet, which can be challenging for patients, especially during adolescence and adulthood [11] - BioMarin emphasizes the importance of monitoring blood Phe levels and dietary intake for effective management of PKU [20]