The FDA has awarded Aeglea's lead therapy, pegzilarginase, a breakthrough drug

recently, Aeglea BioTherapeutics announced that the U.S. Food and Drug Administration has awarded the pilot therapy pegzilarginase (polyglycol arginase) for the treatment of arginase 1 deficiency (ARG1-D) breakthrough drug qualification (BTD).
The FDA granted pegzilarginase BTD based on phase I/II studies conducted in ARG1-D patients and data from ongoing Phase II open label extension studies.
Aeglea expects to continue discussions with the FDA in the fourth quarter of 2019 on the Pegzil Arginase project and its next steps.About pegzilarginase
pegzilarginase is an enhanced anthionine enzyme that is enzyme-depleting and consumes arginine. Currently, Aeglea is developing pegzilarginase for the treatment of patients with arginase 1 deficiency (ARG1-D).
ARG1-D can lead to two key metabolic effects: high levels of arginine and arginine-derived metabolites, urea circulation damage. In terms of treatment, the best treatment currently available to reduce plasma arginine is dietary protein restriction, but compliance is a difficult challenge, and poor compliance often leads to disease progression and premature death.
Pegzilarginase is an enzyme replacement therapy used to reduce elevated arginine levels in the patient's blood. Data from the Phase I/II Study and Phase II Open Label Extension Study evaluating the treatment of PATIENT patients with ARG1-D show clinical improvement and a continued decline in plasma arginine.Currently, Aeglea is recruiting patients for a single global critical Phase III PEACE study that aims to assess the efficacy and safety of pegzilarginase over a placebo treatment for 24 weeks, with the main endpoint being a decrease in the relative baseline level of plasma arginine. (Drug Information Network)