FDA Declines Approval of Ultragenyx’s Sanfilippo Gene Therapy Due to Manufacturing Issues
The FDA has declined to approve Ultragenyx's gene therapy, UX111 (formerly ABO-102), for Sanfilippo syndrome type A, citing issues related to manufacturing and facility procedures.12
The decision was communicated through a Complete Response Letter (CRL), delaying any potential approval until at least 2026.1
The FDA did not raise concerns about the clinical data, trial conduct, or the product's safety and efficacy; the issues are strictly related to chemistry, manufacturing, and controls.12
Ultragenyx is required to provide additional information and address the manufacturing observations before the application can proceed.12
The company believes the manufacturing issues are addressable and plans to work with the FDA to resolve them as quickly as possible.12
Updated clinical data from current patients will need to be included in any resubmission to the agency.2
Sources:
1. https://www.thepharmaletter.com/biotechnology/ultragenyx-hit-with-fda-delay-for-gene-therapy-targeting-rare-childhood-disorder
2. https://www.investing.com/news/sec-filings/fda-issues-complete-response-letter-for-ultragenyx-gene-therapy-application-93CH-4132628