Rare diseases, Sanfilippo Syndrome
RAREUltragenyx Pharmaceutical Inc.Catalyst overview
Research summary
This catalyst tracks a PDUFA approval decision for UX111 (ABO-102) in Rare diseases, Sanfilippo Syndrome. The expected timing is based on the latest available company guidance and may change.
01/30/26:- BLA resubmitted for UX111; PDUFA decision expected Q3 2026; long-term data at WORLDSymposium 2026. 02/03/26:- Transpher A long-term UX111 data for WORLDSymposium 2026 show durable CSF HS reduction; PDUFA decision remains expected Q3 2026. 04/02/26:- FDA accepted UX111 BLA for review; PDUFA action date set for September 19, 2026. 05/05/26:- FDA accepted resubmitted UX111 BLA; long-term data up to eight years show durable clinical improvements and acceptable safety. 08/04/26:- UX111 PDUFA action date reiterated for September 19, 2026.
Verification
Confirm timing and program details against the company announcement or filing.