Drug updated on 9/4/2024
Dosage Form | Injection (subcutaneous; 2.5 mg/0.5 mL, 10 mg/0.5 mL, 20 mg/mL) |
Drug Class | Phenylalanine (Phe)-metabolizing enzymes |
Ongoing and Completed Studies | ClinicalTrials.gov |
Indication
- 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.
Latest News
Summary
- 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.
Product Monograph / Prescribing Information
Document Title | Year | Source |
---|---|---|
Palynziq (pegvaliase-pqpz) Prescribing Information. | 2020 | BioMarin Pharmaceutical Inc., Novato, CA |
Randomized Controlled Trials
Document Title | Sex Distribution | Year | Source |
---|---|---|---|
Pegvaliase for the treatment of phenylketonuria: Results of a long-term phase 3 clinical trial program (PRISM). | Data not availableSubjects F: null% M: null% | 2018 | Molecular Genetics and Metabolism |
Pegvaliase for the treatment of phenylketonuria: A pivotal, double-blind randomized discontinuation Phase 3 clinical trial. | Data not availableSubjects F: null% M: null% | 2018 | Molecular Genetics and Metabolism |
Document Title
Sex Distribution:
Year:
2018
Source:Molecular Genetics and Metabolism
Document Title
Sex Distribution:
Year:
2018
Source:Molecular Genetics and Metabolism
Clinical Practice Guidelines
Document Title | Year | Source |
---|---|---|
Italian national consensus statement on management and pharmacological treatment of phenylketonuria. | 2021 | Orphanet Journal of Rare Diseases |
Evidence- and consensus-based recommendations for the use of pegvaliase in adults with phenylketonuria. | 2019 | Genetics in Medicine |