Ultragenyx's (RARE) pipeline candidate setrusumab shows sustained reductions in fracture rates in patients with osteogenesis imperfecta in new data from the Orbit study.
Ultragenyx Pharmaceutical Inc. revenues from regulatory approved drugs in Q1 of 2024 were $109 million, representing sales growth of 8% compared to the same time period in 2023. Setrusumab is an anti-sclerostin monoclonal antibody being developed for the treatment of patients with osteogenesis imperfecta. DTX-401 is another promising program in the pipeline being developed for the treatment of patients with glycogen storage disease type Ia.
Ultragenyx (RARE) reports meeting primary and secondary endpoints in the late-stage study of its investigational gene therapy, DTX401, to treat glycogen storage disease type Ia.