Aeglea BioTherapeutics has launched Pegzilarginase, an investigational therapy designed to address Arginase 1 Deficiency (ARG1-D), a rare genetic disorder.
Pegzilarginase is an enhanced human arginase developed as an enzyme replacement therapy to reduce elevated levels of arginine in the blood of patients with ARG1-D.
The company is currently enrolling patients for a randomized, three-phase clinical trial to evaluate the safety and effectiveness of pegzilarginase in individuals with ARG1-D.
Positive results from a phase 1/2 trial showed a significant decrease in plasma arginine levels after 8 weeks of treatment with pegzilarginase, and this reduction was sustained over time.
Overall, the therapy was well-tolerated, although a few serious adverse events such as hypersensitivity and hyperammonemia were observed.
Recognizing the urgent need for effective treatments for severe or life-threatening conditions, the US Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation to expedite the development and review process for pegzilarginase.
ARG1-D is an autosomal recessive disorder caused by a mutation that leads to a deficiency of the arginase enzyme. This deficiency impairs the breakdown of arginine, resulting in the accumulation of arginine and ammonia in the bloodstream.
Symptoms of ARG1-D typically become apparent around the age of three and can include developmental delay, seizures, delayed growth, intellectual disability, balance and coordination difficulties, muscle tightness, poor appetite, weak muscle tone, irritability, temperature regulation issues, vomiting, breathing difficulties, small head size, and hyperactivity.
ARG1-D is a debilitating disease associated with progressive neurological abnormalities and early mortality. The introduction of Pegzilarginase offers hope for improved management and treatment outcomes for individuals affected by this condition.
