Hemophilia A Drug Approved by FDA, With Warning

The U.S. Food and Drug Administration approved emicizumab-kxwh, previously known as ACE910, as a once-weekly injection for adults and pediatric patients with hemophilia A who have developed inhibitors. The approval comes with a black box warning about the risk of blood clots because of some patients on emicizumab-kxwh having developed thrombosis while being treated for breakthrough bleeds.

During clinical trials, emicizumab-kxwh reduced the bleed rate by 87 percent in patients who had developed inhibitor-mediated resistance to standard treatment, compared with those who instead received bypassing agents that are now standard therapy.

Roche, the drug’s manufacturer, is still awaiting phase III trial data on emicizumab-kxwh in patients without inhibitors.

Source: Reuters, November 16, 2017.