U.S. Food and Drug Administration Approves BioMarin's PALYNZIQ® (pegvaliase-pqpz) for Adolescents 12 Years of Age and Older with Phenylketonuria (PKU)

Core Insights - The FDA has approved BioMarin Pharmaceutical's supplemental Biologics License Application for PALYNZIQ to include pediatric patients aged 12 and older with phenylketonuria (PKU), making it the only enzyme substitution therapy for this condition [1][5][29] Group 1: FDA Approval and Study Results - The approval is based on the Phase 3 PEGASUS study, which demonstrated that PALYNZIQ significantly reduced blood phenylalanine (Phe) levels compared to diet alone in adolescents aged 12 to less than 18 with uncontrolled blood Phe concentrations greater than 600 µmol/L [2][3] - Participants in the PALYNZIQ group showed a mean reduction in blood Phe levels from 1025 µmol/L at baseline to 567 µmol/L at Week 72, while the diet-only group had a reduction from 1029 µmol/L to 973 µmol/L [3] - By the end of Part 1 of the study, 44.4% of participants reached blood Phe levels below guideline recommendations, with an average reduction of 828 µmol/L, representing a 94% reduction from baseline [3] Group 2: Clinical Implications and Patient Management - PALYNZIQ allows for an unrestricted diet while effectively managing blood Phe levels, which is particularly beneficial for adolescents facing increased independence and academic demands [2][5] - The treatment has shown higher adherence rates in adolescents living at home with family support, making it a promising option for this demographic [2] - The most common adverse reactions in adolescents included injection site reactions, arthralgia, headache, and hypersensitivity reactions, with a notable safety profile observed during the maintenance phase [4][22] Group 3: Company Background and Future Directions - BioMarin has been actively working with medical and advocacy communities for over two decades to improve the lives of individuals with PKU, and the expansion of PALYNZIQ's approval is a continuation of this effort [5][28] - The company is also pursuing approval from the European Medicines Agency to extend PALYNZIQ treatment to adolescents aged 12 and older in the European Union [5][29] - BioMarin is recognized as a leading biotechnology company focused on rare diseases, with a strong pipeline of therapies aimed at genetically defined conditions [28]