Clinical Trials

Date: 2017-11-20

Type of information: Results

phase: 3

Announcement: results

Company: Roche (Switzerland)

Product: emicizumab (ACE910 - anti-factor IXa x anti-factor X humanized bispecific antibody)

Action mechanism:

bispecific antibody. Emicizumab is a bispecific antibody that mimics coagulation factor VIII. It is currently investigated as a therapy for people with hemophilia A. Emicizumab was designated as a Breakthrough Therapy by the FDA in September 2015.

Disease: hemophilia A

Therapeutic area: Rare diseases - Genetic diseases - Hematological diseases

Country: Australia, Costa Rica, France, Germany, Ireland, Italy, Japan, Poland, South Africa, Spain, Taiwan, UK, USA

Trial details:

HAVEN 3 is a randomized, multicenter, open-label, phase III clinical trial to evaluate the efficacy, safety, and pharmacokinetics of prophylactic emicizumab versus no prophylaxis in hemophilia A patients without inhibitors. (NCT02847637)

Latest news:

• On November 20, 2017, Roche announced positive results from the phase III HAVEN 3 study evaluating Hemlibra® (emicizumab) in adults and adolescents (aged 12 years or older) with haemophilia A without inhibitors to factor VIII. The study met its primary endpoint, showing a statistically significant and clinically meaningful reduction in the number of treated bleeds over time in people receiving Hemlibra® prophylaxis every week compared to those receiving no prophylaxis. The study also met key secondary endpoints, including a statistically significant and clinically meaningful reduction in the number of treated bleeds over time with Hemlibra® prophylaxis dosed every two weeks compared to no prophylaxis. Importantly, once-weekly Hemlibra® prophylaxis was superior to factor VIII prophylaxis, as demonstrated by a statistically significant and clinically meaningful reduction in treated bleeds in an intra-patient comparison of patients receiving Hemlibra prophylaxis compared to their prior factor VIII prophylaxis. The most common adverse events with Hemlibra® were injection site reactions, with no new safety signals observed. No thrombotic microangiopathy or thrombotic events occurred in this study. • On July 26, 2016, a Phase 3 trial sponsored by Roche was published on the NIH website ClinicalTrials.gov for and is currently recruiting participants.