Posted by Michael Wonder on 02 Oct 2018
FDA grants rare paediatric disease designation to pegzilarginase for arginase 1 deficiency
1 October 2018 - Aeglea eligible to receive a priority review voucher.
Aeglea BioTherapeutics today announced the U.S. FDA has granted rare paediatric disease designation to the Company’s lead product candidate, pegzilarginase, for the treatment of arginase 1 deficiency. This designation by the FDA confirms Aeglea’s eligibility to receive a rare paediatric disease priority review voucher upon approval of a biologics license application for pegzilarginase. Arginase 1 deficiency is a rare debilitating disease presenting in childhood with persistent hyperargininemia, severe progressive neurological abnormalities and early mortality.
Aeglea recently announced positive interim clinical data for its Phase 1/2 clinical trial of pegzilarginase in arginase 1 deficiency.
