FDA Accepts BLA for Ultragenyx's Gene Therapy for Sanfilippo Syndrome
The FDA has accepted Ultragenyx's (RARE) Biologics License Application (BLA) for UX111, a gene therapy for Sanfilippo syndrome Type A. The PDUFA action date is set for September 19, 2026. If approved, UX111 would be the first treatment for this rare, fatal genetic disorder affecting children.
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Topics: biotech FDA approval gene therapy rare diseases Sanfilippo syndrome