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Aeglea Submits BLA To FDA For Pegzilarginase To Treat Arginase 1 Deficiency

Aeglea BioTherapeutics Inc. (AGLE), a clinical-stage biotechnology company for rare metabolic diseases, Tuesday said it has submitted a Biologics License Application or BLA to the U.S. Food and Drug Administration for pegzilarginase to treat Arginase 1 Deficiency or ARG1-D.

Aeglea requested FDA Priority Review at the time of the BLA submission. If approved, pegzilarginase would be the first FDA-approved treatment for ARG1-D, a rare, progressive disease characterized by high levels of arginine.

Immedica Pharma AB, Aeglea's commercialization partner for pegzilarginase in certain countries in Europe and the Middle East, plans to submit the Marketing Authorization Application or MAA to the European Medicines Agency in 2022.

People living with ARG1-D experience severe spasticity-related mobility limitations, seizures, developmental delay, intellectual disability, and early mortality.

Pegzilarginase is a novel, recombinant human arginase 1 enzyme. In clinical trials, it has been shown to normalize the elevated levels of the amino acid arginine in patients with ARG1-D.

Aeglea's BLA submission includes data from multiple clinical studies in ARG1-D, including the double-blind, placebo-controlled PEACE Phase 3 study and its ongoing long-term extension, a Phase 1/2 clinical trial and an open-label extension study.

Results from these trials demonstrate that pegzilarginase is able to rapidly and sustainably lower arginine levels and showed improvements in mobility.

The FDA has granted pegzilarginase multiple regulatory designations, including Rare Pediatric Disease, Breakthrough Therapy, Fast Track and Orphan Drug designations.

Anthony Quinn, president and chief executive officer of Aeglea, said, "While we have recently received feedback from the FDA expressing disagreement on the substantial evidence of effectiveness of pegzilarginase, we believe based on the totality and compelling nature of our clinical results and the high unmet need that the best path forward for both the ARG1-D community and Aeglea was to proceed with the BLA submission and provide the FDA access to all relevant information."

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