Ultragenyx Pharmaceutical Inc. announced that the FDA has accepted for review its resubmitted Biologics License Application for UX111 gene therapy for Sanfilippo syndrome Type A, with a PDUFA action date of September 19, 2026.
Key takeaways
FDA accepted for review the resubmitted Biologics License Application for UX111 gene therapy for Sanfilippo syndrome Type A.
The FDA set a Prescription Drug User Fee Act action date of September 19, 2026.
The company is seeking accelerated approval for UX111.
The press release contains forward-looking statements about the development, regulatory review, and potential approval of UX111.
Forward-looking statements involve substantial risks and uncertainties that could cause actual results to differ materially.