Ultragenyx Pharmaceutical Inc.
0 products tracked · names used in filings: Ultragenyx Pharmaceutical Inc.
Upcoming milestones
No upcoming milestones listed.
Recent milestones
UX111: PDUFA target action date (Sanfilippo syndrome type A (MPS IIIA))
Ultragenyx Pharmaceutical Inc. (RARE); rebisufligene etisparvovec; BLA; status: set; "…UX111 (rebisufligene etisparvovec) AAV9 gene therapy for the treatment of Sanfilippo syndrome type A (MPS IIIA): In April 2026, the FDA accepted for review the resubmitted BLA seeking accelerated approval for UX111 as a treatment for MPS IIIA and assigned a PDUFA action date of September 19, 2026."; filed 2026-08-04 · source
DTX401: PDUFA target action date (glycogen storage disease type Ia (GSDIa))
Ultragenyx Pharmaceutical Inc. (RARE); pariglasgene brecaparvovec; BLA; status: set; "Food and Drug Administration (FDA) accepted for review the Biologics License Application (BLA) seeking approval of DTX401 as a treatment for GSDIa and assigned a Prescription Drug User Fee Act (PDUFA) action date of August 23, 2026."; filed 2026-08-04 · source
Products
No products recorded.
Information from official EMA, FDA, Federal Register and SEC records. Dates marked company-announced come from the company's own SEC filing; dates marked estimate are calculated, not official. Not investment or medical advice; always confirm on the source.