BLAINJECTIONINJECTABLEPriority Review
Approved
May 2018
Lifecycle
Peak
Competitive Pressure
30/100
Clinical Trials
7
Data completeness
5/8 — Partial
Priority Review
PALYNZIQ (pegvaliase-pqpz) by BioMarin Pharmaceutical is pegvaliase-pqpz is a pegylated phenylalanine ammonia lyase (pal) enzyme that converts phenylalanine to ammonia and trans -cinnamic acid. Approved for phenylketonuria. First approved in 2018.
Drug data last refreshed 19h ago · AI intelligence enriched 3w ago
AI summarySynthesized from FDA + DailyMed + ClinicalTrials.gov data
PALYNZIQ (pegvaliase-pqpz) is a PEGylated phenylalanine ammonia lyase enzyme administered by injection that reduces blood phenylalanine concentrations in adults and older adolescents with phenylketonuria (PKU). It works by substituting for deficient phenylalanine hydroxylase activity, converting phenylalanine to ammonia and trans-cinnamic acid. This represents the first enzyme replacement therapy approved for PKU, addressing a rare genetic metabolic disorder previously managed primarily through dietary restriction.
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Mechanism of Action
Pegvaliase-pqpz is a PEGylated phenylalanine ammonia lyase (PAL) enzyme that converts phenylalanine to ammonia and trans -cinnamic acid. It substitutes for the deficient phenylalanine hydroxylase (PAH) enzyme activity in patients with PKU and reduces blood phenylalanine concentrations.
Pharmacologic Class:
Phenylalanine Metabolizing Enzyme
Indications (1)
Clinical Trials (7)
Immune Modulation During Palynziq® Treatment in Adults (IMPALA)
Started Mar 2026
12 enrolled
Phenylketonuria
Rapid Drug Desensitization Study in Adults Experiencing Hypersensitivity Reactions to Palynziq
Started Feb 2025
9 enrolled
PhenylketonuriaPKU
A Long Term, Post-marketing Study of Immune Response in Patients Receiving Palynziq Treatment for PKU (PALisade)
Started Jan 2024
200 enrolled
Phenylketonuria (PKU)
A Global, Multicenter Study to Assess Maternal, Fetal and Infant Outcomes of Exposure to Palynziq® (Pegvaliase) During Pregnancy and Breastfeeding
Started Nov 2022
50 enrolled
Phenylketonuria, Maternal
A Long-term, Post-marketing Safety Study of Palynziq in Patients With PKU (PALace)
Started Jun 2022
450 enrolled
Phenylketonuria (PKU)
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Data Sources
- Drug label: DailyMed / FDA
- Clinical trials: ClinicalTrials.gov
- Patent data: FDA Orange Book
- Spending data: CMS Medicare
The information on this page is for informational purposes only and should not be used as a substitute for professional medical advice. Drug information is sourced from FDA, DailyMed, and other government databases. Adverse event data from FAERS does not establish causation. Always consult a healthcare professional for medical decisions.