AEB 1102 filed with FDA for Arginase 1 Deficiency.
Aeglea BioTherapeutics has submitted a Biologics License Application (BLA) to the FDA for AEB 1102 (pegzilarginase) for the treatment of Arginase 1 Deficiency (ARG1-D).
Aeglea requested FDA Priority Review at the time of the BLA submission.
Aeglea's BLA submission includes data from multiple clinical studies in ARG1-D, including the double-blind, placebo-controlled PEACE Phase III study and its ongoing long-term extension, a Phase I/II 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.
In the PEACE study, most treatment-emergent adverse events were mild or moderate in severity and there were no discontinuations due to treatment-emergent adverse events. The FDA has granted pegzilarginase multiple regulatory designations, including Rare Pediatric Disease, Breakthrough Therapy, Fast Track and Orphan Drug designations.
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