22 December 2016
Roche has announced that the primary endpoint has been met for the phase III HAVEN 1 study assessing emicizumab prophylaxis in people with haemophilia A and inhibitors to factor VIII. The study revealed a statistically significant reduction in the number of bleeds over time in people treated with emicizumab prophylaxis compared to those receiving no prophylactic treatment. All secondary endpoints were also met, including a statistically significant reduction in the number of bleeds over time with emicizumab prophylaxis treatment in an intra-patient comparison in people who had received prior bypassing agent prophylaxis treatment. Injection site reactions were the most common adverse events with emicizumab, as consistent with prior studies.
Sandra Horning, MD, Chief Medical Officer and Head of Global Product Development, said: “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. 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.”
“Since the mid-1990’s, there have been incremental improvements in the treatment of haemophilia A with inhibitors. The current burden of treatment is significant. WFH is supportive of research that could yield new therapeutic agents and offer a new treatment option for inhibitor patients. Filling this need would be a significant advance in our quest to achieve Treatment for All including those living with inhibitors,” said Alain Baumann, Chief Executive Officer of the World Federation of Hemophilia.