AstraZeneca’s Ultomiris shows strong OS in HSCT TMA

16 June 2025

Initial results from the ALXN1210-TMA-314 Phase III trial showed that UK pharma major AstraZeneca’s (LSE: AZN) Ultomiris (ravulizumab) demonstrated clinically-meaningful overall survival at 26 weeks in pediatric patients (aged 28 days to <18 years of age) with thrombotic microangiopathy (TMA) after hematopoietic stem cell transplantation (HSCT).

The data were presented today at the European Hematology Association (EHA) 2025 Congress in Milan, Italy.1

Data from the global, Phase III, open-label, single-arm study showed that overall survival, a secondary endpoint, was 92.6% (38/41; 95% confidence interval [CI]: 78.8-97.6) at day 100 and 87.2% (36/41; 95% CI: 71.8-94.5) at week 26.

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