U.S. FDA Accepts Ultragenyx UX111 BLA
Analysis based on 8 articles · First reported Apr 02, 2026 · Last updated Apr 02, 2026
The acceptance of the BLA resubmission for UX111 by the United States — Food and Drug Administration is a positive development for Ultragenyx, potentially leading to the first approved therapy for Sanfilippo syndrome Type A. This could significantly boost Ultragenyx's stock price and market position in rare disease therapeutics.
The United States — Food and Drug Administration has accepted the resubmitted Biologics License Application (BLA) from Ultragenyx for UX111 (rebisufligene etisparvovec) AAV9 gene therapy, a potential treatment for Sanfilippo syndrome Type A. This rare, fatal neurodegenerative disorder currently has no approved treatment. The United States — Food and Drug Administration has set a Prescription Drug User Fee Act (PDUFA) action date for September 19, 2026. If approved, UX111 would be the first therapy for this condition and will be manufactured in the United States at facilities including Andelyn Biosciences. The BLA resubmission includes updated long-term clinical data, showing durable treatment effects and an acceptable safety profile. Emil D. Kakkis, CEO of Ultragenyx, expressed optimism about bringing this treatment to patients.
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