7 October 2024 - Ultragenyx today announced that it has received breakthrough therapy designation from the US FDA for setrusumab (UX143) as a treatment to reduce the risk of fracture associated with osteogenesis imperfecta type I, III, or IV in patients 2 years of age and older.

The FDA’s decision is based on preliminary clinical evidence including the positive 14 month results from the Phase 2 portion of the Orbit study, which demonstrated a rapid and clinically meaningful decrease in fracture rate in patients, and from the completed Phase 2b ASTEROID study.

Read Ultragenyx press release