Ultragenyx rises after FDA clears human trial for rare muscle disease drug

Ultragenyx Pharmaceutical

30/03/2026 13:46

** Shares of drugmaker Ultragenyx Pharmaceutical RARE.O rise 2% to $19.76 premarket

** Co says U.S. FDA cleared its application to start human testing of UX016 for GNE myopathy, a rare inherited muscle disease that causes worsening muscle weakness and disability

** There are no approved treatments for the condition, which affects about 10,000 people in commercially accessible markets, co says

** Co plans an early- to mid-stage trial starting in 2H 2026, enrolling about 24 adults in the United States

** UX016 aims to replace sialic acid, a sugar patients cannot make enough of, and is designed to better reach muscle tissue - RARE

** Trial will run for up to 48 weeks and is funded through patient groups and philanthropic support, co says

** Shares fell ~45% in 2025

 (Reporting by Sahil Pandey in Bengaluru)

 ((Sahil.Pandey@thomsonreuters.com))