Ultragenyx Pharmaceutical Receives FDA Clearance For Its Investigational New Drug Application For UX016 To Treat GNE Myopathy

Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) today announced that the U.S. Food and Drug Administration (FDA) has cleared the Investigational New Drug (IND) application for UX016, an investigational small molecule prodrug of sialic acid (SA) being evaluated as a substrate replacement therapy for GNE myopathy (GNEM). GNEM is a rare, severely debilitating, inherited neuromuscular disorder caused by mutations in the GNE gene that lead to deficient SA production. The UX016 program is externally funded by a patient group through clinical proof-of-concept, including a Phase 1/2 study expected to begin in the second half of 2026.