Ultragenyx resubmits BLA for UX111 gene therapy with FDA

Ultragenyx (RARE) Pharmaceutical announced that it has resubmitted its Biologics License Application, BLA, seeking accelerated approval for UX111 AAV9 gene therapy as a treatment for patients with Sanfilippo syndrome type A to the U.S. Food and Drug Administration, FDA. The submission contains substantial longer-term data on multiple measures of neurologic benefit to support an intermediate clinical endpoint for accelerated approval supported further by CSF heparan sulfate and other biomarker data, as agreed with the FDA during the last clinical review.