Pegzilarginase

Pegzilarginase

Pegzilarginase is a recombinant human enzyme used in the treatment of arginase 1 deficiency, a rare genetic disorder that affects the urea cycle. This enzyme is designed to break down arginine, an amino acid that accumulates in patients with this condition, leading to severe health issues.

Mechanism of Action

Pegzilarginase is a pegylated form of the human enzyme arginase 1. Pegylation refers to the process of attaching polyethylene glycol (PEG) chains to a protein, which can improve its stability and solubility, and reduce its immunogenicity. In patients with arginase 1 deficiency, the native enzyme is either absent or non-functional, leading to the accumulation of arginine and ammonia in the blood. Pegzilarginase acts by converting excess arginine into ornithine and urea, thereby reducing the toxic levels of arginine and ammonia.

Clinical Use

Pegzilarginase is primarily used in the treatment of arginase 1 deficiency, a condition that can lead to hyperargininemia, spasticity, developmental delay, and other neurological symptoms. The administration of pegzilarginase helps to manage these symptoms by lowering arginine levels in the blood.

Administration and Dosage

Pegzilarginase is administered via intravenous infusion. The dosage and frequency of administration depend on the patient's weight, the severity of the condition, and the response to treatment. Regular monitoring of plasma arginine levels is necessary to adjust the dosage appropriately.

Side Effects

Common side effects of pegzilarginase include injection site reactions, headache, nausea, and fatigue. More serious side effects can include hypersensitivity reactions and anaphylaxis. Patients receiving pegzilarginase should be monitored for signs of allergic reactions during and after the infusion.

Research and Development

Pegzilarginase is the result of extensive research into enzyme replacement therapies for urea cycle disorders. Clinical trials have demonstrated its efficacy in reducing plasma arginine levels and improving clinical outcomes in patients with arginase 1 deficiency. Ongoing research aims to further understand its long-term effects and potential applications in other metabolic disorders.

Also see

• Arginase 1 deficiency
• Urea cycle disorders
• Enzyme replacement therapy
• Hyperargininemia