On April 2, 2026, Ultragenyx Pharmaceutical Inc. announced that the U.S. Food and Drug Administration (FDA) has accepted for review the company's resubmitted Biologics License Application (BLA) for UX111 (rebisufligene etisparvovec), a gene therapy aimed at treating patients with Sanfilippo syndrome Type A (MPS IIIA). This acceptance marks a significant milestone for Ultragenyx as it seeks accelerated approval for this innovative treatment. The FDA has set a Prescription Drug User Fee Act (PDUFA) action date of September 19, 2026, which indicates when the FDA is expected to make a decision regarding the approval of UX111. The company is optimistic about the potential of UX111 to address the unmet medical needs of patients suffering from this rare genetic disorder. The acceptance of the BLA is a crucial step in the regulatory process and reflects the FDA's recognition of the importance of this therapy. Ultragenyx's commitment to advancing gene therapies for rare diseases is underscored by this development, and the company is preparing for the next stages of the approval process, including potential discussions with the FDA regarding the data submitted in support of the application. Investors and stakeholders are closely monitoring this situation, as the approval of UX111 could have a substantial impact on the company's future growth and market position in the rare disease space.

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