Aeglea BioTherapeutics, Inc. (NASDAQ:AGLE), a clinical-stage biotechnology company that designs and develops innovative human enzyme therapeutics for patients with rare genetic diseases and cancer, today announced the U.S. Food and Drug Administration (FDA) has granted rare pediatric 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 pediatric disease priority review voucher (PRV) upon approval of a biologics license application (BLA) for pegzilarginase. Arginase 1 Deficiency is a rare debilitating disease presenting in childhood with persistent hyperargininemia, severe progressive neurological abnormalities and early mortality.
“This designation underscores the seriousness of Arginase 1 Deficiency and its devastating impact on patients and families living with this disease,” said Anthony G. Quinn, M.B Ch.B, Ph.D., president and chief executive officer of Aeglea. “We look forward to continued collaboration with the FDA as we advance pegzilarginase into a pivotal study in the first half of 2019.”
Aeglea recently announced positive interim clinical data for its Phase 1/2 clinical trial of pegzilarginase in Arginase 1 Deficiency. In addition, the Company announced that it had fully enrolled its Phase 1/2 clinical trial of pegzilarginase by dosing its fifteenth patient, which exceeded the target enrollment of 10 patients. The Company plans to present additional interim clinical data for the Phase 1/2 trial at the 2018 American Society of Human Genetics (ASHG) Conference in October.
About Rare Pediatric Disease Designation
The FDA grants rare pediatric disease designation for diseases with serious or life-threatening manifestations that primarily affect fewer than 200,000 people in the United States and who are less than 18 years old. Under the FDA's Rare Pediatric Disease Priority Review Voucher program, a sponsor who receives an approval of a new drug application or biologics license application for a product for the prevention or treatment of a rare pediatric disease may be eligible for a voucher, which can be redeemed to obtain priority review for any subsequent marketing application or may be sold or transferred.
About Pegzilarginase in Arginase 1 Deficiency
Pegzilarginase is an enhanced human arginase that enzymatically depletes the amino acid arginine. Aeglea is developing pegzilarginase for the treatment of patients with Arginase 1 Deficiency, a rare debilitating disease presenting in childhood with persistent hyperargininemia, severe progressive neurological abnormalities and early mortality. Pegzilarginase is intended for use as an enzyme replacement therapy in patients to reduce elevated blood arginine levels. The Company’s interim Phase 1/2 data demonstrated clinical improvements and rapid and sustained lowering of plasma arginine in Arginase 1 Deficiency patients.