Summary

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• Palynziq (pegvaliase-pqpz) is indicated to reduce blood phenylalanine concentrations in adult patients with phenylketonuria who have uncontrolled blood phenylalanine concentrations greater than 600 micromol/L on existing management.
• This summary is based on the review of two randomized controlled trial(s). ^[1-2]
• Blood Phe Reduction in PRISM-1 and PRISM-2 Studies: At baseline, the mean blood Phe level was 1232.7 µmol/L. After 12 months, it decreased to 564.5 µmol/L (51.1% reduction), and at 24 months, it further decreased to 311.4 µmol/L (68.7% reduction).
• Blood Phe Concentrations in PRISM-2 Randomized Discontinuation Trial (RDT): At RDT Week 8, the placebo groups saw increases in blood Phe levels by 949.8 µM (20 mg/day) and 664.8 µM (40 mg/day), while the pooled pegvaliase group had a minor mean change of 26.5 µM (P < 0.0001 compared to placebo groups).
• Achievement of Blood Phe Targets in PRISM-1 and PRISM-2 Studies: 68.4% of participants achieved blood Phe ≤600 µmol/L, 60.7% achieved ≤360 µmol/L, and 51.2% achieved ≤120 µmol/L.
• In the PRISM-1 and PRISM-2 studies, 99% of adverse events (AEs) were mild or moderate in severity, with 96% resolving without dose interruption or reduction. The most common AEs included arthralgia (70.5%), injection-site reaction (62.1%), injection-site erythema (47.9%), and headache (47.1%).
• Acute systemic hypersensitivity events were observed in 12 participants (17 events) within the PRISM-1 and PRISM-2 studies; these events were not associated with immunoglobulin E and resolved without sequelae. In the Randomized Discontinuation Trial (RDT) of PRISM-2, the incidence of AEs was generally lower in the pooled placebo group compared to the pooled pegvaliase group.
• There is no population types or subgroups information available in the reviewed studies.