Ultragenyx Resubmits BLA for UX111 Gene Therapy Targeting Sanfilippo Syndrome Type A to FDA on February 02, 2026 Accelerated Approved BLA Blackfoot language gene therapy Mucopolysaccharidosis Type IIIA resubmission Ultragenyx United States Food and Drug Administration UX111 +
FDA Declines Approval of Ultragenyx's Sanfilippo Gene Therapy Due to Manufacturing Issues on July 14, 2025 Approved gene therapy manufacturing issues Mucopolysaccharidosis Type IIIA Regulatory Setback Response Letter Ultragenyx United States Food and Drug Administration UX111 +