Ultragenyx Says FDA Accepts Resubmitted BLA For Review Seeking Accelerated Approval For UX111 AAV9 Gene Therapy As Treatment For Patients With Sanfilippo Syndrome Type A; FDA Sets PDUFA Action Date Of Sept. 19

If 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 as a treatment for patients with Sanfilippo syndrome Type A (MPS IIIA). The FDA set a Prescription Drug User Fee Act (PDUFA) action date of September 19, 2026.