Genentech study analyzing emicizumab for hemophilia A meets primary endpoint

Roche Group member Genentech's primary endpoint for the Phase III HAVEN 1 study to evaluate emicizumab prophylaxis in people 12 years of age or older with hemophilia A and inhibitors to Factor VIII has been met.  
“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,” Dr. Sandra Horning, chief medical officer and head of Global Product Development, said in a news release. “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.”
Data from HAVEN 1, the first Phase III study in the emicizumab clinical development program to report results, will be presented at an upcoming meeting. Following that, the study’s results will be submitted to health authorities across the globe for approval.
“Since the mid-1990’s, there have been incremental improvements in the treatment of hemophilia A with inhibitors,” World Federation of Hemophilia CEO Alain Baumann said. “The current burden of treatment is significant. (We are) supportive of research that could yield new therapeutic agents and offer a new treatment option for inhibitor patients.