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Ultragenyx’s bone disease drug fails to improve fracture rate in late-stage trials

By Thomson Reuters Dec 29, 2025 | 7:35 AM

Dec 29 (Reuters) – Ultragenyx Pharmaceutical said on Monday that its ‍experimental drug for a type of genetic bone disease did not meet the main goal ‌in late-stage ‌studies.

The company was testing the treatment called setrusumab in patients with osteogenesis imperfecta, a ​group of genetic disorders impacting bone ‍metabolism.

The condition ​can result in ​increased bone brittleness, which ‍contributes to a high rate of fractures.

The company said the drug failed to meet ‍the main goal of significantly reducing annualized clinical fracture ‍rate, ‍compared to ​placebo.

Both studies, however, ​achieved ⁠the secondary goals ‌of improvements in bone mineral density, the company said.

(Reporting by Sneha S K; Editing by Shailesh ⁠Kuber)