Ultragenyx Announces FDA Clearance of Investigational New Drug (IND) Application for UX016, a Sialic Acid Prodrug for the Treatment of GNE Myopathy
Ultragenyx Pharmaceutical Inc. announced FDA clearance of its Investigational New Drug (IND) application for UX016, a sialic acid prodrug designed to treat GNE myopathy, a rare neuromuscular disorder. The clearance enables Ultragenyx to begin a Phase 1/2 clinical trial in the second half of 2026, with external patient group funding support. UX016 a…