Summary

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• Pegvaliase-pqpz (Palynziq) is indicated to reduce blood phenylalanine concentrations in adult patients with phenylketonuria who have uncontrolled blood phenylalanine concentrations greater than 600 micromol/L.
• Two Randomized Controlled Trials were reviewed for this analysis.
• Palynziq has shown significant effectiveness in reducing blood Phenylalanine levels, as evidenced by PRISM Phase 3 studies where up to 68.4% of participants achieved target Phe levels and up to 51.2% reached even lower Phe levels.
• Palynziq's safety profile reveals a manageable range of adverse events primarily during the initial six months of treatment including arthralgia, injection-site reactions, and instances of systemic hypersensitivity events.
• While direct comparisons with other management strategies within its therapeutic class are not provided in the studies reviewed, Palynziq demonstrates significant therapeutic advancement for adults with PKU uncontrolled by other strategies due to its documented improvements in blood Phenylalanine levels and associated neuropsychiatric benefits along with a manageable safety profile.
• The studies focus on adult populations with PKU but do not provide detailed subgroup analyses such as age-specific responses or differences based on genetic variations within this population.