India, July 27 -- AstraZeneca's (AZN) rare disease unit, Alexion, has announced results from its Phase III program evaluating Ultomiris (ravulizumab) in patients with thrombotic microangiopathy after haematopoietic stem cell transplant (HSCT-TMA), a rare and life-threatening complication.
HSCT-TMA occurs in fewer than 6,000 patients annually in the U.S. and can cause blood clots, organ damage, and death. Treatment options are limited, making new therapies an urgent need.
In the adults and adolescent study (ALXN1210-TMA-313), Ultomiris did not achieve statistical significance for its primary endpoint of event-free survival at 26 weeks compared to placebo, though a trend toward benefit was observed.
In contrast, the pediatric study (ALXN12...