FDA clarifies path to submission for Ultragenyx's rare disease therapy

FDA has given Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) a clear path to an early NDA submission next year using existing data for UX007 to treat long-chain fatty acid oxidation disorders (LC-FAOD), CFO and EVP Shalini Sharp told BioCentury.

Ultragenyx said Wednesday that the agency accepted its proposal for an early submission, pending a pre-NDA meeting this half. The biotech had to make the case that UX007 -- and not other factors -- led to a significant reduction in major clinical events (MCEs) in a Phase II trial, Sharp said...