SEP 19 2026
Ultragenyx Pharmaceutical — UX111 $RARE
for mucopolysaccharidosis type IIIA (MPS IIIA, Sanfilippo syndrome type A)
Accepted APR 2 2026.
About
Sanfilippo syndrome type A is a rare genetic disease where the body cannot break down certain sugar molecules, causing them to build up and progressively damage the brain and spinal cord. UX111 is a gene therapy that uses a modified virus to deliver a functional copy of the SGSH gene directly into the body's cells. This one-time treatment aims to provide the missing enzyme, potentially slowing or halting the neurological decline that defines this condition.
Pivotal trial
The main objective of this study is to evaluate the efficacy and safety of UX111 for the treatment of MPS IIIA.
Source: ClinicalTrials.gov
Also in development
Drug Watch — recent news on UX111
- FDA decision on UX111 gene therapy for Sanfilippo expected in September - Sanfilippo Syndrome News
- FDA Accepts Ultragenyx (RARE) BLA for Sanfilippo Syndrome Gene Therapy UX111 - finance.yahoo.com
- FDA Accepts Ultragenyx’s New BLA Submission for MPS IIIA Gene Therapy UX111 - CGTLive®
- UX111 gene therapy shows long-term benefit in Sanfilippo type A: Trial data - Sanfilippo Syndrome News
- Ultragenyx (RARE) Announces Positive Long-Term Clinical Data for UX111 Gene Therapy in Sanfilippo Syndrome Type A - finance.yahoo.com