The United States Food and Drug Administration (FDA) has granted breakthrough therapy designation to Switzerland-based Roche for its Hemlibra (emicizumab), it was reported yesterday.

The product is intended to treat people with haemophilia A without factor VIII inhibitors. It is a bispecific factor IXa- and factor X-directed antibody and is said to be a prophylactic (preventative) treatment that can be administered by an injection of a ready-to-use solution under the skin (subcutaneously) once-weekly.

The company has been granted the status based on data from the phase III Haven three study in people 12 years or older with haemophilia A without inhibitors. According to the firm, Hemlibra prophylaxis dosed subcutaneously every week or every two weeks indicated a statistically significant and clinically meaningful decrease in treated bleeds compared to no prophylaxis in the study.

Haven 3 is a randomised, multicentre, open-label and phase III study designed to evaluate the efficacy, safety and pharmacokinetics of Hemlibra prophylaxis versus no prophylaxis (episodic/on-demand factor VIII treatment) in people with haemophilia A without inhibitors to factor VIII. The company recruited 152 patients with haemophilia A (12 years of age or older) who were earlier treated with factor VIII therapy either on-demand or for prophylaxis in the trial.

Roche secured first breakthrough therapy designation for Hemlibra in September 2015.