Aeglea BioTherapeutics’ (NASDAQ:AGLE) pegzilarginase received FDA breakthrough therapy designation for the treatment of arginase 1 deficiency (ARG1-D).
ARG1-D is a rare genetic childhood disease that causes severe progressive neurological abnormalities and early mortality. Pegzilarginase is an enzyme replacement therapy designed to reduce elevated blood arginine levels.
“The emerging clinical data demonstrating favorable effects of pegzilarginase treatment on both plasma arginine levels and important disease manifestations has led to the FDA’s designation of pegzilarginase as a breakthrough therapy,” Anthony Quinn, Aeglea’s president and CEO, said in a statement.
The company plans to discuss the program’s next steps with the FDA in the fourth quarter of 2019.