Ultragenyx Pharmaceutical Developed First-of-its-kind Setrusumab (UX143) for Osteogenesis Imperfecta
Ultragenyx Pharmaceutical has developed setrusumab (UX143), a groundbreaking treatment aimed at reducing the fracture risk associated with osteogenesis imperfecta (OI) Types I, III, or IV in patients aged 2 years and older.
Setrusumab is a fully human monoclonal antibody that targets and inhibits sclerostin, a negative regulator of bone formation. By blocking sclerostin, the treatment aims to enhance new bone formation, increase bone mineral density, and strengthen bones in individuals with OI. In mouse models of OI, anti-sclerostin antibodies have demonstrated the ability to boost bone formation, restore bone mass to normal levels, and enhance bone strength in fracture resistance testing.
Osteogenesis Imperfecta (OI) is a group of genetic disorders that affect bone metabolism. Approximately 85% to 90% of OI cases result from genetic variants in the COL1A1 or COL1A2 genes, leading to reduced or abnormal collagen production and disrupted bone metabolism. These collagen mutations contribute to increased bone brittleness, resulting in a high incidence of fractures.
Ultragenyx has received breakthrough therapy designation for setrusumab (UX143) in the treatment of osteogenesis imperfecta.
