Sign in Subscribe
Stock exchanges

Ultragenyx Pharmaceutical Inc. Receives Complete Response Letter for UX111 Gene Therapy

Strategic Insights

— 1 min read

Ultragenyx Pharmaceutical Inc. has announced that the U.S. Food and Drug Administration (FDA) has issued a Complete Response Letter (CRL) for its Biologics License Application (BLA) for UX111, an AAV gene therapy for patients with Sanfilippo syndrome type A (MPS IIIA). The CRL requests additional information and improvements related to specific aspects of chemistry, manufacturing, and controls (CMC) and observations from recent manufacturing facility inspections. The company believes these observations are readily addressable and not directly related to the quality of the product.

Key Takeaways:

  • The FDA issued a CRL for Ultragenyx Pharmaceutical Inc.'s BLA for UX111 on July 11, 2025.
  • The CRL requests additional information and improvements related to CMC and observations from recent manufacturing facility inspections.
  • The company believes these observations are readily addressable and not directly related to the quality of the product.
  • The FDA acknowledged that the neurodevelopmental outcome data provided to date are robust and the biomarker data provide additional supportive evidence.
  • The CRL did not note any review issues related to the clinical data package or clinical inspections.
  • The company expects to resubmit the BLA and anticipates up to a six-month review period to follow the resubmission.
  • Howard Horn, Executive Vice President, Chief Financial Officer, Corporate Strategy of Ultragenyx Pharmaceutical Inc., signed the report on July 11, 2025.

Statistics:

  • The FDA issued the CRL on July 11, 2025.
  • The company plans to resubmit the BLA within a few months and anticipates up to a six-month review period.
  • The CRL did not note any review issues related to the clinical data package or clinical inspections.

Sources:

  • Sen. Forms and reports > SEC forms > Form 8-K

https://www.sec.gov/Archives/edgar/data/136276/0001193125-25-187943-index.htm

Ultragenyx Pharmaceutical Inc. "Current Report Pursuant to Section 13 or 15(d) of the Securities Exchange Act of 1934."

  • SEC Filing, Ultragenyx Pharmaceutical Inc., "Current Report Pursuant to Section 13 or 15(d) of the Securities Exchange Act of 1934."
  • Private Securities Litigation Reform Act of 1995, HR. 1057 - 1st Session

Read more