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Innovation Observatory > Reports > Drugs > Pegzilarginase for Arginase-1 deficiency

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Pegzilarginase for Arginase-1 deficiency


Endocrine, Nutritional and Metabolic

October 2021

Pegzilarginase is in clinical development for the treatment of patients suffering from arginase-1 (ARG1) deficiency. ARG1 deficiency is a rare inherited disease in which the body is unable to process arginine (a building block of protein) due to the lack of the enzyme arginase in the liver and red blood cells. Arginase breaks down and removes nitrogen from the body. The lack of arginase results in excessive accumulation of nitrogen in the form of ammonia in the blood, and arginine in the blood and cerebrospinal fluid. Arginase deficiency presents in early childhood and usually progresses to severe reflexes (spasticity), loss of ambulation, complete loss of bowel and bladder control, and severe intellectual disability. Treatment is focused on lowering arginine levels and preventing buildup of ammonia in the blood.
Pegzilarginase is a novel modified form of the human enzyme ARG1 designed as an intravenous infusion or subcutaneous injection to rapidly and sustainably lower blood arginine levels. If licensed, pegzilarginase will offer an additional treatment option for patients suffering from arginase-1 deficiency who currently have few effective therapies available.

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