Drug offers cost-effective protection for newborns with genetic bleeding disorder

Severe hemophilia A is a genetic disorder that prevents blood from clotting properly, posing a constant risk of dangerous bleeding episodes. Infants with severe hemophilia A (HA) are at risk of brain bleeds, which can lead to serious long-term brain damage. Emicizumab (Hemlibra) was approved by the FDA in 2018 to prevent and reduce bleeding for patients who suffer from hemophilia A. The HAVEN 7 trial showed that emicizumab is both safe and effective in protecting infants from brain bleeds, although concerns were raised about the affordability of this drug.