Roche’s emicizumab haemophilia drug succeeds in phase III trial

Patients receiving emicizumab showed a statistically significant reduction in bleeds over time compared with those not being treated with it.

The treatment also met secondary goals of reducing bleeds in people who had received prior treatment with a bypassing agent.

Roche said the most common adverse events were injection site reactions, consistent with prior studies.

The phase 3 trial was a randomized, multicenter, open-label study evaluating the efficacy, safety and pharmacokinetics of emicizumab prophylaxis versus no prophylaxis. 

It included 109 patients with hemophilia A (12 years of age or older) with inhibitors to factor VIII, who were earlier treated with episodic or prophylactic bypassing agents.

Roche chief medical officer and head of global product development Sandra Horning said: “The development of inhibitors that render factor VIII replacement less effective, or ineffective, is one of the greatest challenges in the treatment of haemophilia A today, putting patients at high risk for life-threatening bleeds and repeated bleeds that may cause long-term joint damage.

“We are pleased to see that, in our first pivotal trial, emicizumab prophylaxis significantly reduced the number of bleeds over time in people in this difficult-to-treat setting. We look forward to working with health authorities to bring this treatment to the haemophilia community as soon as possible.”

Emicizumab is an investigational bispecific monoclonal antibody designed to bring together factors IXa and X, proteins required to activate the natural coagulation cascade and restore the blood clotting process.

It can be administered by an injection of a ready-to-use solution under the skin (subcutaneously) once weekly.

Chugai Pharmaceutical created emicizumab, which is being co-developed by Chugai, Roche and Genentech.

Image: Site Roche Basel. Photo: courtesy of F. Hoffmann-La Roche Ltd.