RARE News & Analysis

Ultragenyx (RARE) BLA Accepted for Gene Therapy UX111 Approval

Ultragenyx (RARE) announced the FDA accepted its resubmitted Biologics License Application (BLA) for UX111, a gene therapy for Sanfilippo syndrome Type A, on April 2. The FDA has set a PDUFA action date of September 19 for a decision. If approved, UX111 would be the first treatment available for this genetic disorder. The therapy is designed to address the underlying enzyme deficiency causing significant health issues in affected children and has shown durable effects in clinical trials lasting up to 8 years.