Ultragenyx Announces U.S. FDA Acceptance of BLA Resubmission for UX111 AAV Gene Therapy to Treat Sanfilippo Syndrome Type A (MPS IIIA)
Ultragenyx Pharmaceutical Inc. (RARE)
Last ultragenyx pharmaceutical inc. earnings: 2/13 04:01 pm
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Source: GlobeNewswire
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. “The FDA’s acceptance of the BLA for UX111 brings us closer to the possibility of a first-ever therapy for Sanfilippo syndrome Type A—a milestone that we recognize cannot come soon enough for families facing this devastating diagnosis,” sa
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