Ultragenyx Pharmaceutical (RARE) has announced that the FDA has granted Breakthrough Therapy Designation to setrusumab for its potential to reduce the risk of fractures in patients aged 2 and older with osteogenesis imperfecta Types I, III, or IV. This decision was supported by promising preliminary clinical evidence, notably the successful 14-month outcomes from the Phase 2 portion of the Orbit study.
Moreover, setrusumab has been recognized with Orphan Drug Designation both in the United States and the European Union. In addition, it has received the rare pediatric disease designation in the United States and has been included in the European Medicine Agency's Priority Medicines program.
