The European Commission recently approved emicizumab (Hemlibra) to prevent bleeding episodes in people with severe haemophilia A without factor VIII inhibitors.

Roche Products and Chugai Pharma say that emicizumab is the “first new class of medicine to treat these haemophilia A patients in nearly 20 years”.

Emicizumab is already approved for haemophilia A with inhibitors to factor VIII. The new approval was based on the HAVEN 3 study, which showed that emicizumab once weekly or once every two weeks reduced treated bleeds by 96 and 97 per cent (annualised treated bleed rate [ABR] 1.5 and 1.3) respectively compared to factor VIII treatment given on-demand (ABR 38.2).

The most serious adverse reactions in the clinical trials were thrombotic microangiopathy and thrombotic events.