Reports Q4 revenue $164.88M, consensus $158.71M. “We have created a next-generation rare disease company on a pathway to profitability with meaningful revenue growth from multiple global products and a series of potential new drug approvals,” said Emil Kakkis, chief executive officer and president of Ultragenyx (RARE). “The major regulatory and clinical catalysts ahead of us this year are the pending PDUFA decision for our gene therapy to treat Sanfilippo syndrome, submission of our second gene therapy biologics license application in Glycogen Storage Disease Type Ia, readout of our pivotal Phase 3 results in osteogenesis imperfecta, and completion of enrollment in our Phase 3 trial in Angelman syndrome.”
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