BioMarin announces positive pivotal data for Palynziq (pegvaliase-pqpz) in adolescents with phenylketonuria

BioMarin Pharmaceutical Inc. announced that the Phase III  PEGASUS trial evaluating Palynziq (pegvaliase-pqpz) met its primary efficacy endpoint, demonstrating a statistically significant lowering in blood 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. 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.

PEGASUS is a Phase III multi-center open-label randomized controlled study  evaluating the safety and efficacy of Palynziq compared to diet alone in 55 adolescents aged 12-17 with phenylketonuria. (NCT05270837).  The primary endpoints are change in blood Phe concentration and characterization of the safety profile in adolescents. Secondary endpoints include change in total dietary protein intake and pharmacokinetics.  The study is being conducted in two parts: the primary treatment phase ranging from weeks 1-73 (Part 1), and the extension phase (Part 2), which lasts for up to an additional 80 weeks of monitoring for the Palynziq arm and allows for crossover for those in the diet-only arm.

“For more than two decades, BioMarin has made strides for people with PKU – pioneering the first treatment, bringing a meaningful new option in PALYNZIQ, and continuing to innovate as we advance our research pipeline. We are encouraged to see these positive data that build on that legacy and show how PALYNZIQ can make an impact for adolescents as they begin their transition to adult living,” said Dr. Greg Friberg, executive vice president and chief research & development officer at BioMarin. “We look forward to sharing these data with the scientific community and to submitting them to global regulators with the goal of making PALYNZIQ available for younger people living with PKU.”