Ultragenyx submits BLA to FDA for UX111 gene therapy in Sanfilippo syndrome type A

Ultragenyx Pharmaceutical Inc.

Submitted BLA to FDA seeking accelerated approval for UX111 (AAV gene therapy) in Sanfilippo syndrome type A (MPS IIIA).
BLA supported by Transpher A study: rapid, sustained CSF heparan sulfate reduction correlated with improved cognitive development vs. natural history.
Most common adverse events were liver enzyme elevations; majority mild/moderate (Grade 1/2), all resolved.
In 2024, FDA agreed CSF heparan sulfate can serve as surrogate endpoint for accelerated approval.

This headline and bullets were generated automatically by deepseek-v4-flash:cloud@v2 from the public filing. Read the source on SEC.gov before relying on any specific claim. Not investment advice. See methodology for how this pipeline works.