Drug updated on 6/26/2023
| 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
- Palynziq 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.
Product Monograph / Prescribing Information
| Document Title | Year | Source |
|---|---|---|
| Palynziq (pegvaliase-pqpz) Prescribing Information. | 2020 | BioMarin Pharmaceutical Inc., Novato, CA |
Systematic Reviews / Meta-Analyses
| Document Title | Year | Source |
|---|---|---|
| Clinical, pharmacoeconomic, and stakeholder input combined report. | 2023 | CADTH |
| Assessment report: Palynziq. | 2019 | EMA |
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 |