The U.S. Food and Drug Administration (FDA) has granted priority review to Roche’s biosimilar for emicizumab, a monoclonal antibody to treat children and adults with hemophilia A without factor VIII inhibitors.
Their decision was based on results from the phase III HAVEN 3 study, which indicated that patients aged 12 years or older who received emicizumab-kxwh every week or every two weeks experienced, respectively, a 96-percent and 97-percent reduction in bleeds, compared with those who received no prophylaxis (p<0.0001 for both). The findings also demonstrated that weekly prophylactic treatment with emicizumab-kxwh was superior to the current standard of care (factor VIII replacement therapy), with a 68-percent reduction in treated bleeds.
The most common adverse events associated with emicizumab-kxwh included reactions to injections, joint pain, common cold symptoms, respiratory infections, and influenza. No thrombotic events or cases of thrombotic microangiopathy were reported.
The drug was granted breakthrough therapy designation in April, and a final decision is expected in October 2018.
Sources: Reuters, June 5, 2018; Roche press release, June 5, 2018.