AstraZeneca's Ultomiris misses HSCT-TMA target but seeks pediatric approval
AstraZeneca's rare disease drug, Ultomiris, didn't hit its key target in a big late-stage trial for HSCT-TMA, a tough complication after stem cell transplants.
The study missed the mark for event-free survival at 26 weeks, though there were hints it might help.
Despite the setback, AstraZeneca isn't giving up and is pushing ahead to get Ultomiris approved for children.
Ultomiris pediatric trial 87.2% survival
Ultomiris actually performed well in a separate pediatric trial: survival rates were 87.2% at 26 weeks and 73.4% at one year.
These results have AstraZeneca moving forward with regulatory filings for children and talking with health authorities about next steps for adults.
Even with recent challenges, like other drug failures, the company still aims to launch up to 20 new therapies by 2030 and hit $80 billion in annual revenue by 2030.