The US Food and Drug Administration (FDA) has granted accelerated approval to Loargys (pegzilarginase-nbln) for the treatment of patients ages 2 years and older with arginase 1 deficiency (ARG1-D).
ARG1-D is a rare genetic disorder characterized by complete or partial lack of the enzyme arginase. Arginase is one of six enzymes that play a role in the urea cycle. The lack of the arginase enzyme results in hyperargininemia in the blood and cerebrospinal fluid, and can lead to hyperammonemia in the blood as well. Children most often experience spasticity (especially in the lower limbs) but they can also experience seizures, short stature, and intellectual disability.
Pegzilarginase is a novel, recombinant, human arginase-1 enzyme that is designed to rapidly and sustainably lower plasma arginine. This new indication is in conjunction with dietary protein restriction. The accelerated approval is based on reduction of plasma arginine and continued approval may be contingent upon verification and description of clinical benefit in a confirmatory trial.
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To learn more about ARG1-D and other rare metabolic disorders, visit https://checkrare.com/diseases/metabolic-disorders/
