Pegzilarginase is a recombinant human arginase 1 enzyme replacement therapy used for the chronic management of hyperargininemia in patients with arginase 1 deficiency (ARG1-D). By substituting the native manganese cofactor with cobalt to enhance catalytic activity, this agent effectively degrades elevated plasma arginine into ornithine and urea, thereby mitigating the accumulation of toxic metabolites that drive the progressive neurological...
- Mechanism
- Curator reviewed · 4 references
Pegzilarginase is a recombinant modified form of the human enzyme arginase 1 (ARG1). It is modified by substituting cobalt for manganese as a cofactor, which increases the catalytic activity of ARG1, and via covalent binding to polyethylene glycol (PEG), which improves blood circulation time. Following administration, pegzilarginase metabolizes the amino acid arginine to ornithine and urea, thereby lowering and normalizing blood arginine levels in patients with ARG1 deficiency.
Pegzilarginase may also have utility as a chemotherapy, as it inhibits the proliferation of cancer cells that depend on extracellular arginine uptake for their growth.
- Primary indication
- Pegzilarginase is indicated in combination with dietary protein restriction for the treatment of hyperargininemia in adult and pediatric patients 2 years of age and older with arginase 1 deficiency (ARG1-D).Curator reviewed · 1 structured indication
- Formula / weight
- 284000.0 Da (224-344 kDa, on average)
- First approval
- Canada, 2026 · United States, 2026 · European Union, 2024
- Also known as
- Co-ArgI-PEG · Co-ArgI-PEG modified human arginase I · Optimised human arginase I
- Code names
- AEB1102
- Brand names
Resolves to
What you can answer from here — as of March 05, 2026
Which drugs share a target with Pegzilarginase, and which of those have an active Phase 3 trial?
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