FDA 483 - Ultragenyx Pharmaceutical Inc. - April 25, 2025

During an FDA inspection conducted from April 21 to April 25, 2025, at Ultragenyx Pharmaceutical Inc. in Bedford, MA, several critical compliance issues were identified. The inspection, led by Massoud Motamed, focused on the company's gene therapy manufacturing processes.

Key violations included deficiencies in data integrity and documentation practices. Specifically, controlled documents, including batch records and training documentation, were improperly handled and found in areas designated for destruction. Additionally, the use of an uncontrolled logbook for batch record tracking was noted, with discrepancies in record location and status.

Facility maintenance and documentation were also problematic. Observations included damaged flooring in manufacturing areas and unresolved leaks, with no deviations documented to assess potential impacts on the manufacturing environment. Furthermore, a biological safety cabinet was found with incomplete assembly and debris, contrary to cleaning protocols.

Deviation management was inadequate, failing to effectively identify, document, or investigate contamination events and other quality-impacting issues. Repeated minor deviations were not escalated appropriately, indicating a lack of robust oversight.

The FDA requires Ultragenyx to address these issues by implementing corrective actions to ensure compliance with Good Manufacturing Practices (GMP). This includes improving documentation integrity, facility maintenance, and deviation management protocols to prevent recurrence and ensure product quality.