Europe’s Committee for Medicinal Products for Human Use (CHMP) has issued a positive opinion supporting the approval of Hemlibra (emicizumab) for routine prophylaxis of bleeding episodes in hemophilia A patients with factor VIII inhibitors.
Hemlibra is a humanized monoclonal antibody that binds to activated factor IX and factor X to restore the function of missing activated factor VIII, which hemophilia A patients need to effectively clot their blood. Nearly a third of these patients can develop inhibitors to factor VIII replacement therapies, putting them at greater risk of life-threatening bleeds or joint damage from continued bleeding episodes.
“Many people with haemophilia A develop inhibitors, which greatly impacts their ability to treat or prevent bleeds and diminishes their quality of life,” Ireland’s Brian O’Mahony, president of the European Haemophilia Consortium, said in a press 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 CHMP — an agency of the 28-member European Union — recommended Hemlibra in all age groups, based on its superior efficacy compared to other treatments evaluated in two Phase 3 studies, HAVEN 1 (NCT02622321) and HAVEN 2 (NCT02795767).
These studies concluded that with once-weekly, ready-to-use, subcutaneous administration of Hemlibra, patients may find relief from hemophilia A. HAVEN 1 and 2 are part of a robust clinical development program that includes two additional Phase 3 studies (HAVEN 3 and 4).
In the HAVEN 1 study of hemophilia A patients with inhibitors aged 12 years and older, Hemlibra prophylaxis showed a statistically significant 87 percent fewer treated bleeds than no prophylaxis.
Interim results from the HAVEN 2 study showed that 87 percent of hemophilia A children with inhibitors younger than 12 who received Hemlibra prophylactic treatment experienced zero treated bleeds.
In HAVEN 3, researchers saw a drop in the number of treated bleeds over time in both adults and teenagers with hemophilia A without inhibitors who received Hemlibra weekly or once every two weeks. In HAVEN 4, results showed a clinically meaningful control of bleeding in that same group who received Hemlibra prophylaxis once every four weeks.
“This positive CHMP opinion represents a significant step towards bringing this new treatment to people with haemophilia A with inhibitors in Europe,” said Dr. Sandra Horning, chief medical officer at Roche. “Hemlibra has been shown to effectively reduce the frequency of bleeds compared to currently available medicines, and with once-weekly administration by injection under the skin, it could also greatly reduce the treatment administration burden, particularly for young children with haemophilia A with inhibitors and their families.”
Data from these studies also led to the November 2017 approval of Hemlibra for routine prophylaxis by the U.S. Food and Drug Administration to prevent or reduce the frequency of bleeding episodes in hemophilia A patients with factor VIII inhibitors.
More recently, the United Kingdom’s Medicines and Health Regulatory Agency issued a positive opinion under its early access program for Hemlibra, based on data from the Phase 3 clinical program.