BLAINJECTIONINJECTABLEPriority Review
Approved
May 2018
Lifecycle
Peak
Competitive Pressure
30/100
Clinical Trials
7
Data completeness
4/8 — 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. First approved in 2018.
Drug data last refreshed 3h ago · AI intelligence enriched 1w ago
AI summarySynthesized from FDA + DailyMed + ClinicalTrials.gov data
PALYNZIQ (pegvaliase-pqpz) is a PEGylated phenylalanine ammonia lyase enzyme therapy that substitutes for deficient phenylalanine hydroxylase activity in phenylketonuria (PKU) patients. It converts phenylalanine to ammonia and trans-cinnamic acid, reducing blood phenylalanine concentrations. Administered via subcutaneous injection, it represents the first enzyme replacement therapy approved for this rare metabolic disorder.
$0.02BPart D
PeakStable
Product is at peak lifecycle maturity with modest Part D spending (~$20M) reflecting a niche rare disease population; commercial team size likely optimized rather than expanding.
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
Indication data is being enriched from DailyMed and FDA labeling. Check back soon for approved therapeutic uses.
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.