NOVATO, Calif., April 22, 2021 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for rare and ultra-rare diseases, today announced the successful completion of an End-of-Phase 2 (EOP2) meeting with the U.S. Food and Drug Administration (FDA) for the DTX301 ornithine transcarbamylase (OTC) deficiency gene therapy program. The meeting focused on the discussion of the Phase 1/2 data and alignment on Phase 3 design and endpoints.
Based on the outcome of this meeting, Ultragenyx has finalized the Phase 3 study design, which will include a 64-week... More