N Engl J Med
Investigational factor VIII replacement therapy safe and effective in severe hemophilia A
February 2, 2023

In the phase 3 XTEND-1 trial, once-weekly efanesoctocog alfa provided superior bleeding prevention to prestudy prophylaxisis. Prophylaxis for 52 weeks was also associated with improved physical health, pain intensity, and joint health. The drug had an acceptable safety profile; headache and arthralgia were the most common adverse events.
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