FDA accepts Ultragenyx resubmission for rare disease gene therapy UX111
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Ultragenyx Announces U.S. FDA Acceptance of BLA Resubmission for UX111 AAV Gene Therapy to Treat Sanfilippo Syndrome Type A (MPS IIIA)
April 2, 2026 8:30 AM EDTIf approved, UX111 will be the first approved therapy for the treatment of Sanfilippo syndrome Type A, a rare disease affecting young children that leads to progressive, irreversible neurodegeneration and early death
PDUFA action date set for September 19, 2026
NOVATO, Calif., April 02, 2026 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) today announced the U.S. Food and Drug Administration (FDA or the Agency) has accepted for review the resubmitted Biologics License Application (BLA) seeking accelerated approval for UX111 (rebisufligene etisparvovec) AAV9 gene therapy... More
