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Europe approves first therapy for haemophilia A with inhibitors in over 20 years

Published on 01/03/18 at 10:26am

Roche has revealed that its bispecific factor IXa- and factor X-directed antibody Hemlibra (emicizumab) has been approved by the European Commission (EC) for routine prophylaxis of bleeding episodes in people with haemophilia A with factor VIII inhibitors.

The company said that almost a third of severe haemophilia A patients can develop inhibitors to therapies which seek to replace factor VIII, the missing element which causes the characteristic bleeding associated with the condition, which leaves them at greater risk of repeated or life-threatening bleeding episodes and long-term joint damage. According to Roche, this group of patients has a 70% increased risk of death compared to

The EC’s decision was based on data which showed that Hemlibra displayed an 87% reduction in treated bleeds compared to no prophylaxis, and 87% of children under 12 treated with the drug experienced no treated bleeds. The data was derived from two clinical studies which Roche claims stand among the largest of their kind in people with haemophilia A with inhibitors.

Additionally, Roche noted that Hemlibra achieved a 79% reduction compared to previous treatment with bypassing agent (BPA) prophylaxis collected in a non-interventional study prior to enrolment.

"We're delighted that the European Commission has approved Hemlibra, providing people with haemophilia A with inhibitors a new medicine for the first time in over 20 years," said Dr Sandra Horning, Roche's Chief Medical Officer and Head of Global Product Development. "We believe Hemlibra has the potential to make a meaningful difference in the lives of people with haemophilia A with inhibitors, and are committed to working with EU member states to provide access to this important medicine as quickly as possible."

Matt Fellows

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