RARE

UX111 (rebisufligene etisparvovec)

FDA PDUFA Target Action Date · Ultragenyx Pharmaceutical Inc.

Saturday, September 19, 2026

Approved

approved

Note: Full approval as FAYUVI (rebisufligene etisparvovec-hopf) for pediatric MPS IIIA (Sanfilippo syndrome type A); reported in Ultragenyx 8-K dated 2026-09-17. CBER product, not yet in drugs.json. Source: https://www.sec.gov/Archives/edgar/data/0001515673/000151567326000006/rare-20260917.htm

Drug Information

Drug Name: UX111 (rebisufligene etisparvovec) (CT.gov)
Application Type: BLA
FDA Designations: Priority Review
Indication: Sanfilippo syndrome type A (mucopolysaccharidosis type IIIA, MPS IIIA)
Approved As: UX111 (rebisufligene etisparvovec)

Filing Details

Company: Ultragenyx Pharmaceutical Inc. →
Stock Ticker: RARE
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

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Data sourced from SEC EDGAR filings. Last updated: 2026-08-02. Report an issue