The company reported positive results from phase III Haven 3 study assessing Hemlibra prophylaxis administered every week or every two weeks in people with haemophilia A without factor VIII inhibitors and the phase III Haven 4 study assessing Hemlibra prophylaxis administered every four weeks in people with haemophilia A with or without factor VIII inhibitors.
According to the company, the adults and adolescents aged 12 years or older without factor VIII inhibitors who secured Hemlibra prophylaxis every week or every two weeks demonstrated a 96% and 97% reduction in treated bleeds in the phase III Haven 3 study compared to those who received no prophylaxis.
Hemlibra is a bispecific factor IXa- and factor X-directed antibody. It has been developed to bring together factor IXa and factor X, which are proteins needed to activate the natural coagulation cascade and restore the blood clotting process for people with haemophilia A.
Roche chief medical officer and global product development head Dr Sandra Horning said: “These new pivotal data show that Hemlibra controlled bleeds in people with haemophilia A, while offering the flexibility of less frequent subcutaneous dosing options.”
Based on Haven 3 study, the US Food and Drug Administration (FDA) granted breakthrough therapy status to Hemlibra for people with haemophilia A without factor VIII inhibitors in April this year.
Haven 3 is a randomised, multicentre, open-label and phase III study assessing the efficacy, safety and pharmacokinetics of Hemlibra prophylaxis versus no prophylaxis in people with haemophilia A without factor VIII inhibitors.
The company recruited 152 patients with haemophilia A, who were previously treated with factor VIII therapy either on-demand or for prophylaxis in the trial.
Haven 4 is a single-arm, multicentre, open-label and phase III study assessing the efficacy, safety and pharmacokinetics (PK) of subcutaneous administration of Hemlibra dosed every four weeks.
The firm enrolled 48 patients with haemophilia A with or without factor VIII inhibitors, who were previously treated with either factor VIII or bypassing agents, on-demand or as prophylaxis.