Pegvaliase-pqpz

(Palynziq®)

Palynziq®

Drug updated on 5/17/2024

Dosage FormInjection (subcutaneous; 2.5 mg/0.5 mL, 10 mg/0.5 mL, 20 mg/mL)
Drug ClassPhenylalanine (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.

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.

Product Monograph / Prescribing Information

Document TitleYearSource
Palynziq (pegvaliase-pqpz) Prescribing Information.2020BioMarin Pharmaceutical Inc., Novato, CA

Randomized Controlled Trials

Document TitleSex DistributionYearSource
Pegvaliase for the treatment of phenylketonuria: Results of a long-term phase 3 clinical trial program (PRISM).
Data not availableSubjects
F: null%
M: null%
2018Molecular 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%
2018Molecular Genetics and Metabolism

Sex Distribution:

No Data

Year:

2018

Source:Molecular Genetics and Metabolism

Clinical Practice Guidelines