Aeglea Bio Gets Refusal to File Lette r from FDA

 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 received a Refusal to File (RTF) letter from the U.S. Food and Drug Administration (FDA) regarding the company's Biologics License Application (BLA) for pegzilarginase for the treatment of Arginase 1 Deficiency (ARG1-D).

In the RTF letter the FDA requested additional data to support effectiveness, such as evidence showing that plasma arginine and metabolite reduction predicts clinical benefit in patients with ARG1-D or clinical data demonstrating a treatment effect on clinically meaningful outcomes. The FDA also requested additional information relating to Chemistry Manufacturing and Controls (CMC). There were no issues related to safety raised in the letter. Aeglea intends to request a Type A meeting with the FDA to clarify and respond to the items identified in the RTF letter.

https://finance.yahoo.com/news/aeglea-biotherapeutics-receives-refusal-file-125900337.html