Regulatory Affairs

Roche’s Hemlibra gets FDA breakthrough status in haemophilia A without inhibitors

PBR Staff Writer Published 18 April 2018

Roche has secured breakthrough therapy designation from the US Food and Drug Administration (FDA) for its Hemlibra (emicizumab) to treat people with haemophilia A without factor VIII inhibitors.

Hemlibra is a bispecific factor IXa- and factor X-directed antibody. It 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 received status based on data from the phase III Haven 3 study in people 12 years or older with haemophilia A without inhibitors.

According to the company, Hemlibra prophylaxis dosed subcutaneously every week or every two weeks demonstrated a statistically significant and clinically meaningful reduction in treated bleeds compared to no prophylaxis in the study.

Haven 3 is a randomised, multicentre, open-label and phase III study designed to assess 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.

Roche recruited 152 patients with haemophilia A (12 years of age or older) who were previously treated with factor VIII therapy either on-demand or for prophylaxis in the trial.

Roche chief medical officer and global product development head Dr Sandra Horning said: “Hemlibra is the first medicine to show superior efficacy compared to factor VIII prophylaxis, the standard of care for people with haemophilia A without inhibitors, in an intra-patient comparison.

“We look forward to working with health authorities to make Hemlibra available to people without inhibitors as soon as possible, and we are excited to share this news with the community as we join in celebrating World Hemophilia Day.

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

In November 2017, Hemlibra received FDA approval for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adults and children with haemophilia A with factor VIII inhibitors.

The approval was based on data from the Haven 1 and Haven 2 studies.

In February this year, Hemlibra secured approval from the European Commission for routine prophylaxis of bleeding episodes in people with haemophilia A with factor VIII inhibitors.