Regulatory Affairs

Roche's hemophilia drug Hemlibra secures approval in Europe

PBR Staff Writer Published 28 February 2018

Roche’s Hemlibra (emicizumab) has been approved by the European Commission (EC) as a prophylactic treatment for bleeding episodes in patients with hemophilia A with factor VIII inhibitors.

The drug is a bispecific antibody that targets the factor IXa- and factor X proteins.

Hemlibra has been designed to bind the two proteins, which are needed to trigger the natural coagulation cascade and repair the blood clotting process in haemophilia A patients.

The drug is indicated to be injected subcutaneously, once in a week, and can be used in all age groups.

Created by Chugai Pharmaceutical, Hemlibra has been co-developed by the Japanese company in partnership with Roche and Genentech.

The European approval of Hemlibra has been driven by the results from two phase 3 studies Haven 1 and Haven 2.

Hemlibra had shown superior efficacy, in comparison to prior treatment with bypassing agents (BPA) as prophylaxis or on-demand in the two late-stage trials.

In both the trials, which were held in people with haemophilia A with inhibitors, Hemlibra showed its ability to substantially reduce bleeds in adults and children. While Haven 1 was held in 109 patients, aged 12 years or over, Haven 2 featured 23 children, aged under 12 years.

Roche chief medical officer and global product development head Sandra Horning said: "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.

"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."

The European approval of Hemlibra comes on the heels of its approval in the US in November 2017. Like in Europe, the approval for Hemlibra in the US is for routine prophylaxis to prevent or decrease frequency of bleeding incidents in patients with hemophilia A with factor VIII inhibitors.


Image: Roche’s Hemlibra has been approved for treating bleeding in certain hemophilia A patients. Photo: courtesy of Business Wire.