European Commission OKs Hemlibra to Preventively Treat Hemophilia A with Factor VIII Inhibitors

The European Commission has approved Hemlibra (emicizumab) for routine preventive treatment of bleeding episodes in people who have hemophilia A with factor VIII inhibitors.

The antibody therapy is designed to restore the factor VIII, which is deficient in hemophilia A.

Hemlibra is being co-developed by Japan’s Chugai Pharmaceuticals, Switzerland’s Roche, and Roche subsidiary Genentech.

“Following the U.S. approval, we are very proud of the approval to deliver the first-in-class product Hemlibra to people with hemophilia A with inhibitors in the EU as well,” Tatsuro Kosaka, Chugai’s president and chief operations officer, said in a press release.

The approval follows a Committee for Medicinal Products for Human Use (CHMP) positive opinion of Hemlibra adopted in January and a U.S. Food and Drug Administration (FDA) approval in November 2017.

Both the CHMP positive opinion and the European Medicines Agency (EMA) approvals are based on two Phase 3 studies — HAVEN1 (NCT02622321) for adolescents and adults, and HAVEN2 (NCT02795767) in children. The clinical program also includes HAVEN 3 (NCT02847637) and HAVEN 4 (NCT03020160).

Combined, the four studies showed that Hemlibra prophylactic (preventive) treatment led to a statistically significant 87 percent reduction in treated bleeds than seen with no prophylaxis; about 87 percent of hemophilia A pediatric patients who received Hemlibra prophylactic treatment experienced no treated bleeds. There was a progressive decrease in the number of treated bleeds over time, and Hemlibra also led to a meaningful control of bleeding in patients who received it once every four weeks.

In a 59th American Society of Hematology (ASH) Annual Meeting presentation in February, the HAVEN clinical program was continuing to show significant reductions in bleeding episodes in patients with hemophilia A.

Updated results reportedly show that in hemophilia A patients with inhibitors to clotting factor VIII, prophylaxis with once-weekly subcutaneous (under-the-skin) injections of Hemlibra is significantly more effective than prior treatment with bypassing agents (BPAs), both as prophylaxis or as on-demand treatment. The company is now studying less-frequent dose regimens of Hemlibra.

About a third of hemophilia A patients can develop inhibitors to factor VIII replacement therapies, putting them at greater risk of life-threatening bleeds or joint damage from long-term bleeding episodes.

Hemlibra brings together factors IXa and X, normalizing the clotting cascade, so it potentially can be effective on both patients who develop factor VIII inhibitors and those who do not.