Ultragenyx Receives Complete Response Letter from FDA for UX111 AAV Gene Therapy to Treat Sanfilippo Syndrome Type A (MPS IIIA)
Ultragenyx Pharmaceutical(US:RARE) Globenewswire·2025-07-12 04:30Complete Response Letter (CRL) cited specific chemistry, manufacturing and controls (CMC) related observations that are resolvable FDA clinical reviews acknowledged that the clinical data are robust and biomarker data are supportive NOVATO, Calif., July 11, 2025 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), today announced that the U.S. Food and Drug Administration (FDA) has issued a Complete Response Letter (CRL) for its Biologics License Application (BLA) for UX111 (ABO-102) AAV gene ...