NasdaqGS:RAREBiotechs
How UX111’s FDA Resubmission and First-in-Disease Potential Will Impact Ultragenyx Pharmaceutical (RARE) Investors
Ultragenyx Pharmaceutical recently resubmitted its Biologics License Application to the FDA for UX111, an investigational AAV9 gene therapy for Sanfilippo syndrome type A, supported by new long-term clinical data showing durable biomarker improvements, meaningful functional benefits versus natural history, and a favorable safety profile.
An interesting aspect for investors is that UX111, if approved after the current FDA review expected to conclude in the third quarter of 2026, would become...