OCT 2 2026
Ultragenyx Pharmaceutical — UX111 $RARE
for mucopolysaccharidosis type IIIA (MPS IIIA, Sanfilippo syndrome type A)
Accepted APR 2 2026.
About
Mucopolysaccharidosis type IIIA (Sanfilippo syndrome type A) is a rare genetic disorder where the body cannot properly break down certain sugar molecules, leading to their buildup and progressive damage to the brain and spinal cord. UX111 is a gene therapy that uses a virus to deliver a functional copy of the SGSH gene into cells, aiming to restore the missing enzyme needed to break down these sugars. By addressing the root cause of the disease, UX111 may potentially slow or prevent the neurological decline associated with 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 - Yahoo Finance
- FDA Accepts Ultragenyx’s New BLA Submission for MPS IIIA Gene Therapy UX111 - CGTLive®
- Ultragenyx Announces U.S. FDA Acceptance of BLA Resubmission for UX111 AAV Gene Therapy to Treat Sanfilippo Syndrome Type A (MPS IIIA) - Yahoo Finance
- Ultragenyx BLA for UX111 Accepted for FDA Review - TipRanks