RARE News & Events

Ultragenyx announced the FDA has accepted for review the resubmitted biologics license application seeking accelerated approval for UX111 AAV9 gene therapy as a treatment for patients with Sanfilippo syndrome Type A. The FDA set a Prescription Drug User Fee Act action date of September 19.

Ultragenyx announced that the FDA has cleared the investigational new drug application for UX016, an investigational small molecule prodrug of sialic acid being evaluated as a substrate replacement therapy for GNE myopathy. The UX016 program is externally funded by a patient group through clinical proof-of-concept, including a Phase 1/2 study expected to begin in the second half of 2026.