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On February 23, 2026, the FDA approved Loargys (pegzilarginase-nbln) from Immedica Pharma for hyperargininemia in adults and pediatric patients 2 years and older with Arginase 1 Deficiency, in conjunction with dietary protein restriction. ARG1-D affects an estimated 250 people in the U.S.A.
Immedica Pharma’s CEO, Anders Edvell, said, of the approval, “Today’s FDA accelerated approval of Loargys is an important milestone for Immedica and for patients and families affected by ARG1-D in the U.S.”
Pegzilarginase-nbln is a recombinant human arginase-1 enzyme replacement therapy.
The FDA granted accelerated approval based on reduction of plasma arginine at Week 24, with 90.5% of treated patients below the guideline target for plasma arginine in the Phase 3 PEACE trial (NCT03921541), sponsored by Aeglea BioTherapeutics before the asset was acquired by Immedica Pharma in 2023.
Dr. Stephen Cederbaum, Professor Emeritus of Human and Medical Genetics at UCLA highlighted that, “Until now, the care of patients with ARG1-D has been limited to symptomatic management and strict dietary control. The accelerated approval of Loargys offers a fundamentally new approach that addresses the enzyme deficiency itself.”
Clinically, it establishes a labeled enzyme replacement therapy option for Arginase 1 Deficiency in the approved population.
Sources: FDA Novel Drug Approvals for 2026 ; Immedica press release ; ClinicalTrials ; Efficacy and safety of pegzilarginase in arginase 1 deficiency (PEACE): a phase 3, randomized, double-blind, placebo-controlled, multi-centre trial; Immedica press release on acquisition of pegzilarginase
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