pegzilarginase-nbln (Loargys)

Indications

• enzyme therapy for treating hyperargininemia in patients >=2 years with arginase 1 deficiency (ARG1-D)
  • to be used in conjunction with dietary protein restriction

• lowers high plasma arginine levels that cause severe neurological decline, including spasticity & developmental delays.

Dosage

• administered weekly via subcutaneous injection: 5 MG/ML 0.4 mL, 1 mL

Adverse effects

• injection site reactions, itching, skin rashes, vomiting, hyperammonemia

Laboratory

• prior to initiating treatment, a baseline plasma arginine concentration should be obtained
• after initiating treatment, weekly

Mechanism of action

• recombinant human arginase 1 enzyme (ARG1) that breaks down arginine into ornithine & urea.

Notes

• Price/Cost (2016):
  • one unit of Loargys (2 mg/0.4 mL) listed at $11,211.12 to $28,027.80