The manufacturers of the bi-specific antibody emicizumab reported the death of a patient with hemophilia with inhibitors who was enrolled in the HAVEN 1 trial. HAVEN 1 is an ongoing phase III clinical trial evaluating the safety and efficacy of emicizumab in patients ≥12 years with hemophilia A and factor VIII inhibitors. The monoclonal antibody works by binding to activated factors IX and X, a normal function of factor VIII that is diminished in hemophilia A.
The European Haemophilia Consortium (EHC) received report of the death and requested an investigation from the drug manufacturer, Roche, to evaluate safety and adverse events (AEs). The patient who died was reported to have two serious AEs: a rectal hemorrhage and a thrombotic microangiopathy. The hemorrhage was reported to be the cause of death and was not considered to be treatment-related.
Based on this report, the EHC recommended avoiding the use of activated prothrombin complex in the trial and using the lowest approved doses of bypassing agents in this patient population.
Source: European Haemophilia Consortium press release, February 21, 2017.