Ultragenyx and Mereo BioPharma announced that the Phase 3 Orbit study of setrusumab for osteogenesis imperfecta (OI) in pediatric and young adult patients is proceeding towards its final analysis, expected by the end of 2025. A Data Monitoring Committee confirmed the drug’s acceptable safety profile, recommending the study’s continuation. Concurrently, the Phase 3 Cosmic study, focusing on younger OI patients, is also progressing well with a consistent safety profile.
This continued development of setrusumab is crucial for OI patients, as there are currently no globally approved treatments for this debilitating genetic disorder affecting approximately 60,000 individuals in commercially accessible regions. Positive results from these studies could offer the first approved therapy targeting the underlying causes of OI, potentially improving bone strength, reducing fracture rates, and enhancing quality of life for these patients.
Both Orbit and Cosmic studies are evaluating setrusumab’s effectiveness in reducing fracture rates. Orbit, enrolling patients aged 5 to 25, has completed enrollment with 183 participants. Cosmic, an active-controlled study, is comparing setrusumab to intravenous bisphosphonates in 69 patients aged 2 to under 7. Final analyses for both studies are planned after patients complete at least 18 months of treatment, using a p-value threshold of <0.04 for Orbit and <0.05 for Cosmic. The anticipated final analyses of Orbit and Cosmic represent a significant milestone in the development of setrusumab. Positive results could lead to regulatory submissions and potential approval of the first disease-modifying treatment for OI, marking a substantial advance in the field and offering new hope for patients and their families.
Jon Napitupulu is Director of Media Relations at The Clinical Trial Vanguard. Jon, a computer data scientist, focuses on the latest clinical trial industry news and trends.
