Post by Ultragenyx

Name: Today we announced the U.S. FDA has accepted for review the resubmitted Biologics License Applicatio
Uploaded: 2026-04-02T12:42:29.501Z
Channel: Ultragenyx
Description: Today we announced the U.S. FDA has accepted for review the resubmitted Biologics License Application (BLA) for an investigational gene therapy for Sanfilippo syndrome Type A (MPS IIIA). The FDA set a

116,023 followers

Today we announced the U.S. FDA has accepted for review the resubmitted Biologics License Application (BLA) for an investigational gene therapy for Sanfilippo syndrome Type A (MPS IIIA). The FDA set a Prescription Drug User Fee Act (PDUFA) action date of September 19, 2026. If approved, it would be the first therapy for the treatment of Sanfilippo syndrome Type A, an ultra-rare disease affecting young children that leads to progressive, irreversible neurodegeneration and early death. View the full announcement: https://lnkd.in/gUd7YCjq

Video Content