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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
NOVATO, Calif., June 12, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) today announced that the company held a successful meeting with the U.S. Food and Drug Administration (FDA or the Agency) during which the company reached agreement with the Agency that cerebral spinal fluid (CSF) heparan sulfate (HS) is a reasonable surrogate endpoint that could support submission of a biologics license application (BLA) seeking accelerated approval for UX111 (ABO-102) AAV gene therapy for the treatment of Sanfilippo syndrome (MPS IIIA). The company will need to finalize details of its BLA with the Agency in a pre-BLA meeting with the intent to file late this year or early next.
Posted In: RARE
