Summary

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• This summary is based on the review of two randomized controlled trial(s). ^[1-2]
• In the PRISM-1 and PRISM-2 studies, treatment with pegvaliase resulted in a mean reduction of blood phenylalanine (Phe) levels from a baseline of 1232.7 (386.4) µmol/L to 564.5 (531.2) µmol/L at 12 months (51.1% reduction) and further to 311.4 (427) µmol/L at 24 months (68.7% reduction), with 68.4% of participants achieving blood Phe ≤600 µmol/L and 60.7% achieving blood Phe ≤360 µmol/L.
• Improvements in neuropsychiatric outcomes were observed and sustained over the long-term treatment with pegvaliase, indicating a correlation between reduced blood Phe levels and enhanced neuropsychiatric function in adults with phenylketonuria (PKU) who had baseline blood Phe levels >600 µmol/L.
• The studies did not identify significant differences in effectiveness among various population types or subgroups, focusing primarily on adults with PKU.
• In the PRISM-1 and PRISM-2 studies, adverse events (AEs) were reported to be more frequent during the first 6 months of treatment, with 99% of AEs classified as mild or moderate in severity. Common AEs included arthralgia (70.5%), injection-site reactions (62.1%), injection-site erythema (47.9%), and headache (47.1%).
• Serious adverse events included acute systemic hypersensitivity reactions in 12 participants (17 events), with 6 of these participants continuing treatment. These hypersensitivity events were not associated with immunoglobulin E and resolved without sequelae.
• There is no population types or subgroups information available in the reviewed studies.