- In February 2025, Ultragenyx Pharmaceutical Inc. announced that the U.S. Food and Drug Administration (FDA) has accepted for review its Biologics License Application (BLA) for UX111 (ABO-102), an AAV gene therapy intended to treat patients with Sanfilippo syndrome type A (MPS IIIA), under the accelerated approval pathway. The application was granted Priority Review, with a Prescription Drug User Fee Act (PDUFA) target action date set for August 18, 2025. The FDA also indicated that it does not currently plan to convene an advisory committee meeting to review the application
- In May 2022, Ultragenyx Pharmaceuticals secured licensing rights for an experimental gene therapy aimed at treating Sanfilippo Syndrome. This acquisition strengthens their rare disease pipeline by incorporating innovative treatment options for the condition. The move highlights the company's focus on advancing gene therapy solutions for neurodegenerative disorders
- In January 2022, JCR Pharmaceuticals' drug JR-441 was granted "Orphan Drug" status in Europe for the treatment of Sanfilippo Syndrome. This designation provides the company with additional market exclusivity and regulatory incentives for developing the treatment. The status reinforces JCR's commitment to addressing rare diseases through innovative therapies
