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ROCKVILLE, Md., Aug. 18, 2025 /PRNewswire/ -- REGENXBIO Inc. (NASDAQ:RGNX) today announced that the U.S. Food and Drug Administration (FDA) extended its review timeline of the Biologics License Application (BLA) for clemidsogene lanparvovec (RGX-121) for the treatment of Mucopolysaccharidosis II (MPS II), also known as Hunter syndrome. The Prescription Drug User Fee Act (PDUFA) goal date has been extended from November 9, 2025 to February 8, 2026.
The extension follows the Company's submission of longer-term clinical data for all patients in the pivotal study of RGX-121 (n=13) in response to an FDA information request. These positive 12-month clinical data are consistent with biomarker and neurodevelopmental data previously submitted on the same patients in the BLA and will be presented during the International Congress of Inborn Errors of Metabolism (ICIEM) in September 2025.
In August 2025, the FDA completed a pre-license inspection and bioresearch monitoring information inspection for the RGX-121 BLA with no observations. No safety-related concerns have been raised by the FDA during the BLA review.
"Boys with this rare, devastating disease have no treatment options to address neurodevelopmental decline, and the Hunter syndrome community is in urgent need for a therapeutic option with the potential to improve these patients' lives," said Curran M. Simpson, President and Chief Executive Officer of REGENXBIO. "We promptly provided the FDA with the information requested and expect the commercial launch plans remain on track."
RGX-121 has received Orphan Drug Product, Rare Pediatric Disease, Fast Track and Regenerative Medicine Advanced Therapy (RMAT) designations from the FDA and advanced therapy medicinal products (ATMP) classification from the European Medicines Agency.
About RGX-121 (clemidsogene lanparvovec)
RGX-121 is a potential one-time AAV therapeutic for the treatment of boys with MPS II, designed to deliver the iduronate-2-sulfatase (IDS) gene to the central nervous system (CNS). Delivery of the IDS gene within cells in the CNS could provide a permanent source of secreted iduronate-2-sulfatase (I2S) protein beyond the blood-brain barrier, allowing for long-term cross correction of cells throughout the CNS. RGX-121 expressed protein is structurally identical to normal I2S.
Posted In: RGNX
