UX111 (rebisufligene etisparvovec)
FDA PDUFA Target Action Date · Ultragenyx Pharmaceutical Inc.
Note: Ultragenyx Form 8-K, Item 8.01, filed 2026-04-02: 'the U.S. Food and Drug Administration (the "FDA") has accepted for review the Company's resubmitted Biologics License Application seeking accelerated approval for UX111 (rebisufligene etisparvovec) AAV9 gene therapy as a treatment for patients with Sanfilippo syndrome Type A (MPS IIIA). The FDA set a Prescription Drug User Fee Act (PDUFA) action date of September 19, 2026.' Corroborated in Ultragenyx's Q1 2026 press release filed as 8-K EX-99.1 on 2026-05-05 (https://www.sec.gov/Archives/edgar/data/1515673/000119312526206368/rare-ex99_1.htm): 'In February 2025, the FDA granted the BLA Priority Review and, in April 2026, assigned a PDUFA action date of September 19, 2026.' The filing is a BLA RESUBMISSION seeking accelerated approval. No approval or CRL as of 2026-08-02 (no Ultragenyx 8-K filed after 2026-05-18). This entry is absent from both pdufa-manual.json and pdufa-calendar.json.
Drug Information
- Application Type
- BLA
- FDA Designations
- Priority Review
- Indication
- Sanfilippo syndrome type A (mucopolysaccharidosis type IIIA, MPS IIIA)
Filing Details
- Company
- Ultragenyx Pharmaceutical Inc.
- Stock Ticker
- RARE
- SEC CIK
- Source
- SEC 8-K →
What is a PDUFA Date?
A PDUFA (Prescription Drug User Fee Act) date is the FDA's target deadline to complete review of a drug application. The FDA aims to make an approval decision by this date.
- Standard Review: 10 months from filing acceptance
- Priority Review: 6 months from filing acceptance
- Possible Outcomes: Approval, Complete Response Letter (CRL), or extension
Track All FDA Decisions
View all upcoming PDUFA dates and AdCom meetings on our FDA Calendar.
View FDA Calendar →Data sourced from SEC EDGAR filings. Last updated: 2026-08-02. Report an issue