NasdaqGS:RAREBiotechs
Why Ultragenyx Pharmaceutical (RARE) Is Up 12.2% After FDA Accepts UX111 Gene Therapy BLA Review
Ultragenyx Pharmaceutical recently announced that the FDA accepted for review its resubmitted BLA for UX111, an AAV9 gene therapy for Sanfilippo syndrome Type A, setting a PDUFA action date of September 19, 2026 and acknowledging robust long-term neurodevelopmental and biomarker data with an acceptable safety profile.
This progress, alongside FDA clearance of the IND for UX016 in GNE myopathy, highlights Ultragenyx’s deepening focus on late-stage gene and rare disease therapies backed by...