EU Agency Recommends Emicizumab for Approval

The European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use recommended marketing authorization for emicizumab. Emicizumab is the first monoclonal antibody for the treatment of patients with hemophilia A and inhibitors. The development of this medication represents a significant advance in hemophilia treatment.

“Many people with hemophilia A develop inhibitors, which greatly impacts their ability to treat or prevent bleeds and diminishes their quality of life,” said Brian O’Mahony, chief executive of the Irish Haemophilia Society and president of the European Haemophilia Consortium, in a Roche news release. “It’s been over 20 years since we last saw a new medicine for people with hemophilia A with inhibitors, so there is a pressing need for innovative treatments that control bleeding and decrease the negative impact on quality of life.”

The EMA panel’s recommendation is based on data from two phase III clinical trials: a randomized, open-label trial that included 109 patients aged 12 years or older, and an ongoing single-arm, open-label study that included results for 60 children younger than 12 years.

Overall, when used preventively in patients with hemophilia with inhibitors, emicizumab decreased bleeding episodes requiring treatment with coagulation factors by 87 percent when compared with on-demand use of bypassing medications without prophylactic treatment. The most frequently reported adverse events were injection-site reactions, headache, thrombotic microangiopathy, fever, diarrhea, and joint and muscle pain.

The US Food and Drug Administration approved emicizumab on November 16, 2017.

Sources: European Medicines Agency press release, January 26, 2018; Roche press release, January 26, 2018.