Posted by Michael Wonder on 24 Jul 2019
Aeglea BioTherapeutics receives FDA breakthrough therapy designation for pegzilarginase for treatment of arginase 1 deficiency
24 July 2019 - Designation follows recently reported ARG1-D Phase 1/2 data demonstrating clinical response in patients rreated with pegzilarginase.
Aeglea BioTherapeutics today announced that the U.S. FDA has granted breakthrough therapy designation to the Company’s lead investigational therapy, pegzilarginase, for the treatment of Arginase 1 Deficiency (ARG1-D), a rare progressive disease presenting in early childhood that results in severe complications and early mortality.
For pegzilarginase, breakthrough therapy designation was based on data from the Phase 1/2 and ongoing Phase 2 open-label extension clinical trials in patients with ARG1-D.
