Emicizumab for hemophilia A meets primary endpoint in Phase III study

The primary endpoint has been met for the Phase III HAVEN 1 study evaluating emicizumab (ACE910) prophylaxis in people 12 years of age or older with hemophilia A and inhibitors to factor VIII, the drug’s developer, Swiss pharma giant Roche (ROG: SIX), announced this morning.

The study showed a statistically-significant reduction in the number of bleeds over time in people treated with emicizumab prophylaxis compared to those receiving no prophylactic treatment. The study also met all secondary endpoints, 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. The most common adverse events with emicizumab were injection site reactions, consistent with prior studies.

“The development of inhibitors that render factor VIII replacement less effective, or ineffective, is one of the greatest challenges in the treatment of hemophilia A today, putting patients at high risk for life-threatening bleeds and repeated bleeds that may cause long-term joint damage,” said Sandra Horning, chief medical officer and head of global product development. “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 hemophilia community as soon as possible,” Dr Horning added.