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Pegvaliase-Pqpz
Pegvaliase-pqpz is a pegylated phenylalanine ammonia lyase that catalytically degrades phenylalanine to reduce blood phenylalanine levels in patients with phenylketonuria.
Pegvaliase-pqpz is a pegylated phenylalanine ammonia lyase that catalytically degrades phenylalanine to reduce blood phenylalanine levels in patients with phenylketonuria. Used for Phenylketonuria (PKU) in patients with blood phenylalanine levels ≥600 μmol/L.
At a glance
| Generic name | Pegvaliase-Pqpz |
|---|---|
| Sponsor | University of Missouri-Columbia |
| Drug class | Enzyme replacement therapy |
| Target | Phenylalanine (substrate); phenylalanine ammonia lyase (active enzyme) |
| Modality | Small molecule |
| Therapeutic area | Metabolic/Genetic disorder |
| Phase | FDA-approved |
Mechanism of action
The enzyme phenylalanine ammonia lyase (PAL) converts the amino acid phenylalanine into trans-cinnamic acid and ammonia, effectively lowering circulating phenylalanine concentrations. Pegylation extends the drug's half-life and reduces immunogenicity. This mechanism addresses the underlying metabolic defect in phenylketonuria by providing an alternative pathway for phenylalanine catabolism.
Approved indications
- Phenylketonuria (PKU) in patients with blood phenylalanine levels ≥600 μmol/L
Common side effects
- Injection site reactions
- Anti-drug antibodies/immunogenicity
- Arthralgia
- Headache
Key clinical trials
- A Long-term, Post-marketing Safety Study of Palynziq in Patients With PKU (PALace)
- A Global, Multicenter Study to Assess Maternal, Fetal and Infant Outcomes of Exposure to Palynziq® (Pegvaliase) During Pregnancy and Breastfeeding
- A Long Term, Post-marketing Study of Immune Response in Patients Receiving Palynziq Treatment for PKU (PALisade)
- Immune Modulation During Palynziq® Treatment in Adults (IMPALA) (PHASE4)
- Nutritional Impacts of Palynziq on Patients With Phenylketonuria (PKU)
- Preliminary Study of Brain Effects of Palynziq-Related Changes in Phenylalanine in Individuals With PKU
- Rapid Drug Desensitization Study in Adults Experiencing Hypersensitivity Reactions to Palynziq (PHASE4)
- Nutrition Status of Adults Treated With Pegvaliase
Primary sources
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |
Competitive intelligence
For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:
- Pegvaliase-Pqpz CI brief — competitive landscape report
- Pegvaliase-Pqpz updates RSS · CI watch RSS
- University of Missouri-Columbia portfolio CI