FDA agrees CSF heparan sulfate as surrogate endpoint for UX111 accelerated approval BLA in MPS IIIA

Ultragenyx Pharmaceutical Inc.

FDA agreed cerebral spinal fluid heparan sulfate is a reasonable surrogate endpoint supporting accelerated approval for UX111 gene therapy in Sanfilippo syndrome type A (MPS IIIA).
Company intends to file BLA late 2024 or early 2025 after a pre-BLA meeting with the FDA.
BLA filing will rely on data from the ongoing pivotal Transpher A study and long-term follow-up; results were presented at WORLDSymposium.

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