pegzilarginase at a glance
Medicine class
Pegylated recombinant human arginase enzyme therapy
Licensed use
Arginase 1 deficiency in adults, adolescents and children aged 2 years and older
Administration
Intravenous infusion or subcutaneous injection under specialist supervision
Key monitoring
Plasma arginine testing needs validated sample handling because pegzilarginase can distort routine results
What is pegzilarginase used for?
Licensed use:
Treatment of arginase 1 deficiency, also known as hyperargininaemia, in adults, adolescents and children aged 2 years and older.
Used with individualised disease management, which may include protein restriction, amino-acid supplements and medicines for other metabolic problems.
How does pegzilarginase work?
In arginase 1 deficiency, arginine cannot be adequately broken down. Pegzilarginase replaces missing enzyme activity, converting arginine into urea and ornithine. Its polyethylene glycol component slows enzyme clearance.
pegzilarginase preparations and strengths
Injection
Route: Parenteral
Strengths: 5 mg/mL
How to take pegzilarginase
Pegzilarginase is given by intravenous infusion or subcutaneous injection. A healthcare professional normally administers it. Trained patients or carers may give subcutaneous injections at home after specialist assessment.
Do not shake the vial.
For subcutaneous use, rotate suitable injection sites and avoid scarred, red, inflamed or swollen skin.
Each vial is for single use only.
Keep refrigerated, do not freeze and protect from light.
pegzilarginase side effects
Common or expected effects
- Injection-site pain, redness, swelling, irritation or rash
- Hypersensitivity reactions, including rash, flushing or facial swelling
Get urgent medical advice
- Seek urgent help for throat or tongue swelling, chest tightness, wheezing, breathing difficulty, faintness or collapse.
pegzilarginase in pregnancy
Human pregnancy information is absent or very limited, and animal studies found reproductive toxicity. Pegzilarginase is not recommended during pregnancy or for someone who could become pregnant and is not using contraception. Pregnancy planning or pregnancy during treatment needs prompt specialist review.
pegzilarginase while breastfeeding
It is unknown whether pegzilarginase passes into breast milk, and a risk to a breastfed child cannot be excluded. The specialist and patient should decide whether to stop breastfeeding or interrupt treatment after weighing benefits to both parent and child.
pegzilarginase interactions
Formal interaction studies have not been performed. As pegzilarginase is a recombinant human enzyme, interactions mediated through cytochrome P450 metabolism are not expected.
Common questions about pegzilarginase
Answers are fully visible for fast scanning and source review.
Can children receive pegzilarginase?
It is licensed for children aged 2 years and older, as well as adolescents and adults with arginase 1 deficiency. Safety and effectiveness below 2 years have not been established.
Does pegzilarginase replace a low-protein diet?
No. It is used alongside individualised disease management, which may include dietary protein restriction, amino-acid supplements and other medicines.
Can pegzilarginase be given at home?
Subcutaneous home administration may be considered after specialist assessment, once treatment is stable, allergy risk is judged low and the patient or carer has been trained.
Sources
These sources were used to prepare and review this medicine page.
Prepared and reviewed by the iatroX editorial team.