Ultragenyx gets Complete Response Letter for Sanfilippo syndrome gene therapy

5 hours ago 1
Paper with words Sanfilippo syndrome and glasses.
  • The U.S. FDA has issued a Complete Response Letter to Ultragenyx Pharmaceutical (NASDAQ:RARE) for its BLA for UX111 (ABO-102), its AAV gene therapy to treat Sanfilippo syndrome type A.
  • The company said that the agency cited issues related to chemistry, manufacturing

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