22 December 2016
Prophylaxis with emicizumab reduced the number of bleeds over time compared with no prophylaxis in the HAVEN 1 study, which recruited people aged >12 years with haemophilia A and inhibitors.
Manufacturer Roche said the phase III study had also met its 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.
“Since the mid-1990s, there have been incremental improvements in the treatment of haemophilia A with inhibitors,” said Alain Baumann, Chief Executive Officer of the World Federation of Hemophilia. “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 programme 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.