Hemlibra (emicizumab) has been approved in Europe for people with severe haemophilia A without factor VIII inhibitors, Roche has announced.
Hemlibra can be used in all age groups and can also now be used at multiple dosing options for all indicated people with haemophilia A, including those with factor VIII inhibitors.
The approval is based on results from the pivotal HAVEN 3 and HAVEN 4 studies.
In the HAVEN 3 study in people with haemophilia A without factor VIII inhibitors, Hemlibra prophylaxis led to statistically significant and clinically meaningful reductions in treated bleeds compared to no prophylaxis, and compared to prior treatment with factor VIII prophylaxis in a prospective intra-patient comparison.
In the HAVEN 4 study in people with haemophilia A with and without factor VIII inhibitors, Hemlibra showed a clinically meaningful control of bleeding when dosed every four weeks.
“Today’s approval is a landmark moment as Hemlibra is the first new class of treatment for people with severe haemophilia A without inhibitors in nearly 20 years,” said Sandra Horning, Roche’s Chief Medical Officer and Head of Global Product Development.
“Moreover, Hemlibra can effectively control bleeds while offering subcutaneous dosing once weekly, every two weeks or every four weeks. We will continue to work with EU member states, to bring this important treatment to those in need as quickly as possible.”