Hemophilia A Treatment Meets Primary Endpoint in Phase III

Genentech’s emicizumab for hemophilia A meets primary endpoint in phase III study.

Genentech, a member of the Roche Group, announced that the primary endpoint has been met for the phase III HAVEN 1 study evaluating emicizumab prophylaxis in people 12 years of age or older with hemophilia A and inhibitors to factor VIII.

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, M.D., 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.”

Alain Baumann, chief executive officer of the World Federation of Hemophilia, said that since the mid-1990s there have been incremental improvements in the treatment of hemophilia A with inhibitors and that 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.”

As previously reported, two patients had thromboembolic events and two patients developed thrombotic microangiopathy (TMA). The common aspect between all cases of thromboembolic events and TMA is that they occurred in patients who were on emicizumab prophylaxis and in addition received activated prothrombin complex concentrate to treat breakthrough bleeds. Neither thromboembolic event required anti-coagulation therapy and one patient restarted emicizumab. Both cases of TMA have completely resolved, and one patient restarted emicizumab.

HAVEN 1 is the first phase III study in the emicizumab clinical development program to report results. Data from the study will be presented at an upcoming medical meeting and submitted to health authorities around the world for approval consideration.

(Source: Business Wire)