Ultragenyx Pharmaceutical Inc.
UX111
Sanfilippo syndrome Type A (MPS IIIA)
Countdown
75days
FDA decision · Sep 19, 2026
What's at stake
The FDA's approval decision on UX111 for Sanfilippo syndrome Type A (MPS IIIA) is due Sep 19, 2026. A green light opens the market; a complete response letter (CRL) usually sinks the stock.
Primary source
· SEC 8-K
verified 13h ago
“the resubmitted BLA included substantial longer-term data that were presented in February at the WORLD Symposium™ 2026 and included up to eight years of follow-up. These data demonstrated further clinical improvement relative to the decline”