Ultragenyx Pharmaceutical Inc. (RARE) on Thursday said the U.S. Food and Drug Administration has accepted its resubmitted Biologics License Application seeking accelerated approval for UX111, an AAV9 gene therapy for the treatment of Sanfilippo syndrome Type A.
The FDA set a Prescription Drug User Fee Act action date of September 19, 2026.
Sanfilippo syndrome Type A is a rare, fatal neurodegenerative disorder with no approved treatment, primarily affecting the brain and causing rapid decline beginning in early childhood.
The company said that during its prior late-cycle review, the FDA acknowledged the robustness of neurodevelopmental outcomes and noted that biomarker data provided additional supportive evidence.
Ultragenyx added that updated long-term clinical data, including up to eight years of follow-up and presented at WORLDSymposium 2026, showed sustained clinical benefit compared with natural disease progression, along with a durable treatment effect across multiple biomarkers and an acceptable safety profile.
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May 15, 2026 15:25 ET Apart from the confirmation of Kevin Warsh as the next Fed chair, the main news on the economics front this week included key price data from the U.S. and the first quarter economic growth figures from major economies. Both consumer prices and producer costs have started to reflect the effect of supply shocks due to the Middle East conflict. In Europe, GDP data was in focus, while inflation data from China dominated the news flow in Asia.