BioMarin Advances PALYNZIQ Treatment for Adolescents with Phenylketonuria (PKU)

BioMarin Advances Treatment for Phenylketonuria in Adolescents

BioMarin Pharmaceutical Inc. has made a significant stride in expanding treatment options for adolescents suffering from phenylketonuria (PKU) with the acceptance of its supplemental Biologics License Application (sBLA) for PALYNZIQ® (pegvaliase-pqpz) by the U.S. Food and Drug Administration (FDA). This application, now under Priority Review, aims to broaden access to this innovative enzyme substitution therapy for patients aged 12 to 17. The FDA has set a Prescription Drug User Fee Act (PDUFA) target action date of February 28, 2026, signaling a potential milestone in the regulatory process for this critical therapy.

The impetus for the sBLA stems from promising outcomes reported in the Phase 3 PEGASUS study, which involved 55 adolescents and aimed to assess the safety and efficacy of PALYNZIQ. The trial demonstrated a statistically significant reduction in blood phenylalanine (Phe) levels when compared to dietary management alone. With primary endpoints focused on changes in blood Phe concentration and safety profile characterization, the PEGASUS study substantiates BioMarin's commitment to providing effective therapeutic solutions for managing PKU in younger populations. The trial's structure, which includes a primary treatment phase and an extension phase, also allows for continued monitoring and potential crossover for participants who initially received diet-only treatment.

Greg Friberg, M.D., BioMarin's Executive Vice President and Chief R&D Officer, underscores the urgent need for improved treatment modalities for adolescents with PKU. Many of these patients struggle to achieve recommended Phe levels while maintaining dietary freedom. As the first and only approved enzyme substitution therapy for adult PKU treatment, PALYNZIQ represents a groundbreaking approach to managing this metabolic disorder, potentially redefining the therapeutic landscape for adolescents as well. BioMarin is concurrently collaborating with the European Medicines Agency to extend PALYNZIQ's approval for the same age group in Europe, further demonstrating its commitment to addressing this rare condition on a global scale.

In addition to its regulatory efforts, BioMarin remains focused on advancing research and development initiatives. The company continues to explore innovative therapies while gathering real-world data to enhance patient outcomes. As it progresses with PALYNZIQ, BioMarin aims to solidify its position as a leader in the treatment of rare genetic disorders, prioritizing the needs of patients and their families across various age groups.

For more information on the clinical trials and ongoing research, interested parties can visit BioMarin's dedicated clinical trials website.