#United States #San Rafael #BioMarin #PKU #PALYNZIQ

FDA Grants Priority Review for PALYNZIQ® (pegvaliase-pqpz)

BioMarin Pharmaceutical Inc. has made a significant advancement in the treatment of phenylketonuria (PKU) with the U.S. Food and Drug Administration (FDA) accepting its Supplemental Biologics License Application (sBLA) for PALYNZIQ. This submission aims to expand its use to adolescents aged 12-17, a demographic that has previously faced limited treatment options.

The FDA's designation of Priority Review signifies the agency's commitment to expedite the evaluation of therapies that offer substantial benefits to patients. The target action date for the Prescription Drug User Fee Act (PDUFA) is set for February 28, 2026, highlighting an optimistic timeline for potential approval.

Importance of PALYNZIQ for Adolescents with PKU

Before this development, adolescents with PKU had limited dietary options, needing to adhere to a strict low-phenylalanine diet to manage their condition effectively. Greg Friberg, M.D., Executive Vice President and Chief Research and Development Officer at BioMarin, emphasized the need for innovative treatment options. He stated, “Adolescents and young adults with PKU need better options that can deliver meaningful reductions in blood Phe levels and provide greater dietary freedom.” The results derived from the Phase 3 PEGASUS study indicate that PALYNZIQ could significantly lower blood phenylalanine (Phe) levels while allowing patients to consume higher quantities of protein from whole foods.

PALYNZIQ, previously approved solely for adult patients with PKU, is particularly revolutionary due to its enzyme substitution mechanism compared with diet alone, making it an essential addition to the treatment options for teens. The PEGASUS trial showcased PALYNZIQ's ability to maintain lower blood Phe levels effectively, thus providing hope to families navigating diet management in typical adolescent life.

Study Details and Implications

The PEGASUS study involved 55 adolescents between the ages of 12 and 17 and measured both safety and efficacy against dietary restrictions alone. Observing both primary and secondary endpoints clarified how this treatment can significantly affect the lives of those managing PKU. The study, which was presented at the 15th International Congress of Inborn Errors of Metabolism in Kyoto, Japan, is crucial in providing robust data supporting this expansion of treatment.

BioMarin’s commitment extends beyond the United States; they are also working with the European Medicines Agency to introduce PALYNZIQ for adolescent patients within the European Union. This international effort is aimed at providing life-altering therapies more broadly to those affected by PKU worldwide.

The Science Behind PALYNZIQ

PALYNZIQ works by substituting the deficient enzyme, phenylalanine hydroxylase (PAH), with a PEGylated version that efficiently breaks down phenylalanine in the blood, preventing the toxic accumulation that leads to severe neurological consequences if untreated. Patients receiving this therapy will have a structured dosage regimen designed to improve tolerability while being closely monitored for safety concerns. Notably, administration may carry a risk of anaphylaxis, which necessitates careful oversight during treatment.

BioMarin reiterates a strong interest in ensuring that those eligible for the treatment have the necessary support through their RareConnections program, offering personalized assistance for patients and caregivers navigating the complexities of managing a rare metabolic condition.

Conclusion

The FDA's acceptance of BioMarin's application marks a pivotal moment for the PKU community, particularly for adolescents who have been in need of better treatment regimens. As the approval date approaches, the hopes of patients and families hinge on the possibility of a brighter, more flexible future guided by this potential new treatment.

For ongoing updates and further details, visit BioMarin's dedicated clinical trial information page at clinicaltrials.biomarin.com. The commitment BioMarin shows through its research and collaboration with regulatory bodies could set the stage for revolutionary advancements in rare disease treatment, benefitting many in the PKU community and beyond.