Efficacy and safety of pegzilarginase in patients below 2 years of age with arginase 1 deficiency: a phase 3, open-label, multi-centre study.
BACKGROUND: Arginase 1 Deficiency (ARG1-D) is a rare metabolic disorder characterized by marked hyperargininaemia and progressive neurological impairment. INTERPRETATION: In patients with ARG1-D <2 years of age, pegzilarginase demonstrated pharmacokinetic and pharmacodynamic responses comparable to older children and a favourable safety profile.
BACKGROUND: Arginase 1 Deficiency (ARG1-D) is a rare metabolic disorder characterized by marked hyperargininaemia and progressive neurological impairment. INTERPRETATION: In patients with ARG1-D <2 years of age, pegzilarginase demonstrated pharmacokinetic and pharmacodynamic responses comparable to older children and a favourable safety profile.
What the study was
- Study design
- Phase 3 clinical trial
- Category
- Drug Development
- Maturity
- Potentially Practice-Changing
- Journal
- EClinicalMedicine
Why it surfaced
High-priority Rare diseases with high unmet need study: novel treatment potential warrants immediate pipeline escalation.
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