Ultragenyx announces completion of enrollment in Phase 3 studies of setrusumab

Ultragenyx Pharmaceutical announced that all patients have been enrolled across the Phase 3 Orbit and Cosmic studies evaluating setrusumab in pediatric and young adult patients with osteogenesis imperfecta. The pivotal Phase 3 portion of the Orbit study has randomized 158 patients ages 5 to 25 years, and the Cosmic study has completed enrollment of 66 patients ages 2 to less than7 years. Data presented at the American Society for Bone and Mineral Research 2023 Annual Meeting from the Phase 2 portion of the Orbit study showed that treatment with setrusumab reduced the median annualized fracture rate by 67% and this reduction was associated with continuing large and meaningful improvements in bone mineral density. Setrusumab was generally well tolerated with no drug-related serious adverse events reported and no reports of drug-related hypersensitivity. Additional longer-term Phase 2 safety and efficacy data from the Orbit study are expected in the second half of 2024.