Clinical TrialJul 27, 2026, 07:11 AM
AstraZeneca Ultomiris Adult HSCT-TMA Trial Misses Primary Endpoint
AI Summary
AstraZeneca's Ultomiris (ravulizumab) Phase III trial (ALXN1210-TMA-313) in adults and adolescents with thrombotic microangiopathy after haematopoietic stem cell transplant (HSCT-TMA) did not meet its primary endpoint of event-free survival through 26 weeks. However, a separate open-label Phase III trial (ALXN1210-TMA-314) in pediatric patients with HSCT-TMA demonstrated clinically meaningful overall survival of 87.2% at 26 weeks and 73.4% at 52 weeks. Alexion, AstraZeneca Rare Disease, is proceeding with regulatory filings for Ultomiris in pediatric HSCT-TMA based on these positive results, while discussions with health authorities are ongoing regarding the adult and adolescent data.
Key Highlights
- Ultomiris Phase III trial (ALXN1210-TMA-313) in adults/adolescents with HSCT-TMA did not achieve statistical significance for primary endpoint.
- Primary endpoint was event-free survival through 26 weeks compared to placebo.
- Pediatric Phase III trial (ALXN1210-TMA-314) showed 87.2% overall survival at 26 weeks and 73.4% at 52 weeks.
- Alexion is advancing regulatory filings for Ultomiris in pediatric HSCT-TMA.
- Ultomiris showed a trend toward treatment benefit in adults/adolescents; discussions with health authorities are ongoing.
- Ultomiris has Orphan Drug Designation in US/Japan and Breakthrough Therapy designation by US FDA for pediatric HSCT-TMA.
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