Roche’s phase III study of haemophilia drug emicizumab prophylacis for 12 yearolds and older has met its primary endpoint, as it showed bleeding reduction in people treated with it, compared to those who weren’t treated.
Roche said that the study also met all secondary endpoints.
Sandra Horning from Roche said that the development of inhibitors that render factor VIII replacement less effective, or ineffective, is one of the greatest challenges in the treatment of haemophilia A today, putting patients at high risk for life-threatening bleeds and repeated bleeds that may cause long-term joint damage.”
She said: “We are pleased to see that, in our first pivotal trial, emicizumab prophylaxis significantly reduced the number of bleeds over time in people in this difficult-to-treat setting. We look forward to working with health authorities to bring this treatment to the haemophilia community as soon as possible.”