Ultragenyx to File for Accelerated Approval of Treatment of Sanfilippo Syndrome

 Agreement reached with FDA that cerebral spinal fluid (CSF) heparan sulfate (HS) can be used as a reasonable surrogate endpoint for accelerated approval

Data from existing clinical studies appear sufficient for a biologics license application (BLA) submission

https://www.globenewswire.com/news-release/2024/06/12/2897865/20739/en/Ultragenyx-Announces-Plans-to-File-for-Accelerated-Approval-of-UX111-for-the-Treatment-of-Sanfilippo-Syndrome-Type-A-MPS-IIIA.html