AstraZeneca’s rare disease drug misses main goal in late-stage trial
Reuters
Mon, July 27, 2026 at 12:31 p.m. GMT+5:30
1 min read
July 27 (Reuters) – AstraZeneca said on Monday its rare disease drug Ultomiris failed a late-stage trial in patients with a blood vessel complication following a stem cell transplant, the latest in a series of setbacks this year that have fuelled concerns about the drugmaker’s pipeline
• Ultomiris did not achieve statistical significance for event-free survival at 26 weeks in patients with haematopoietic stem cell transplant-associated thrombotic microangiopathy, though it showed a trend toward benefit
• AstraZeneca said it is in ongoing discussions with global health authorities over potential next steps for the adult indication
• In a separate trial involving children, Ultomiris showed an overall survival rate of 87.2% at 26 weeks and 73.4% at 52 weeks, and the drugmaker said it will advance regulatory filings for use in children
• The data comes on the heels of a failed pivotal heart-disease trial earlier this month, which have cast doubts over the drugmaker’s trial design
• In May, a U.S. regulatory panel rejected its breast cancer drug camizestrant on trial design grounds
• AstraZeneca also reported that its experimental gastric cancer drug sonesitatug vedotin met a key goal, showing a statistically significant and clinically meaningful improvement in overall survival in advanced gastric cancer patients
• The drugmaker, which topped second-quarter profit expectations on strong demand for its cancer and rare disease therapies, is counting on up to 20 new drug launches to help generate $80 billion in annual revenue by 2030
• Ultomiris, known chemically as ravulizumab, is already approved in the United States, the EU and Japan for treating certain rare blood disorders and neurological conditions
(Reporting by Raechel Thankam Job in Bengaluru; Editing by Mrigank Dhaniwala and Nivedita Bhattacharjee)
