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Aeglea BioTherapeutics Submits BLA to FDA for Pegzilarginase for the Treatment of Arginase 1 Deficiency
BLA submission provides FDA with all pegzilarginase program data to review in detail; Aeglea looks forward to working collaboratively with the FDA on next
About this update from Spyre Therapeutics, Inc.
[{"type":"text","content":"BLA submission provides FDA with all pegzilarginase program data to review in detail; Aeglea looks forward to working collaboratively with the FDA on next steps in the review process\nIf approved, pegzilarginase would be the first FDA-approved treatment for this devastating rare disease\nEuropean marketing application on track for submission this year\nAUSTIN, Texas, April 12, 2022 /PRNewswire/ -- Aeglea BioTherapeutics, Inc. (Nasdaq:AGLE), a clinical-stage biotechnology company developing a new generation of human enzyme therapeutics to benefit people with rare metabolic diseases, today announced that it has submitted a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for pegzilarginase for the treatment of Arginase 1 Deficiency (ARG1-D). Aeglea requested FDA Priority Review at the time of the BLA submission.\nPegzilarginase is a novel, recombinant human arginase 1 enzyme that in clinical trials has been shown to normalize the elevated levels of the amino acid arginine in patients with ARG1-D, a rare, progressive disease characterized by high levels of arginine. People living with ARG1-D experience severe spasticity-related mobility limitations, seizures, developmental delay, intellectual disability, and early mortality.\n\"The submission of our first BLA is a significant achievement in the evolution of Aeglea, but more importantly, it is an important step forward for the ARG1-D community. While we have recently received feedback from the FDA expressing disagreement on the substantial evidence of effectiveness of pegzilarginase, we believe based on the totality and compelling nature of our clinical results and the high unmet need that the best path forward for both the ARG1-D community and Aeglea was to proceed with the BLA submission and provide the FDA access to all relevant information,\" said Anthony G. Quinn, M.B., Ch.B., Ph.D., president and chief executive officer of Aeglea. \"On behalf of Aeglea, I'd like to thank all the patients and families, investigators, staff and advocates who have helped us achieve this important milestone. I would also like to thank our employees who have worked incredibly hard over the last few months preparing the BLA to enable this submission. We look forward to continuing to work with the FDA through the review of our BLA and are excited about the potential ...