REGENXBIO Inc. announced on August 18, 2025, that the U.S. Food and Drug Administration (FDA) extended its review timeline for the Biologics License Application (BLA) for clemidsogene lanparvovec (RGX-121) for Mucopolysaccharidosis II (MPS II).
The Prescription Drug User Fee Act (PDUFA) goal date has been extended from November 9, 2025, to February 8, 2026. This extension follows the company's submission of longer-term clinical data for all 13 patients in the pivotal study of RGX-121 in response to an FDA information request.
The FDA completed a pre-license inspection and bioresearch monitoring information inspection for the RGX-121 BLA in August 2025 with no observations, and no safety-related concerns have been raised during the BLA review. The submitted 12-month clinical data are consistent with previously submitted biomarker and neurodevelopmental data.
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