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April 02, 2026

Ultragenyx Announces U.S. FDA Acceptance of BLA Resubmission for UX111 AAV Gene Therapy to Treat Sanfilippo Syndrome Type A (MPS IIIA)

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

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March 30, 2026

Ultragenyx Announces FDA Clearance of Investigational New Drug (IND) Application for UX016, a Sialic Acid Prodrug for the Treatment of GNE Myopathy

Program to be externally funded by a venture philanthropy agreement through clinical proof-of-concept, including the Phase 1/2 study planned to begin in the second half of 2026 NOVATO, Calif., March 30, 2026 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc.

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