The US FDA Accepts Ultragenyx’s Resubmitted BLA for UX111 in Sanfilippo Syndrome Type A

Shots:

• The US FDA has accepted the resubmitted BLA of UX111 (rebisufligene etisparvovec) for accelerated approval in Sanfilippo syndrome Type A (MPS IIIA), with a PDUFA action date of Sep 19, 2026
• UX111 BLA is supported by up to 8yrs. of follow-up data showing durable clinical benefit & acceptable safety. Also, during prior late-cycle review, the FDA acknowledged robust neurodevelopmental outcomes with biomarker data providing additional supportive evidence
• If approved, Ultragenyx is planning US-based manufacturing via Andelyn Biosciences in Columbus, Ohio & via its gene therapy manufacturing facility in Bedford, Massachusetts

Ref: Ultragenyx |  Image: Ultragenyx  |Press Release

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