Roche Announces HAVEN 3 & HAVEN 4 Study Results

Roche announced full results from the phase III HAVEN 3 study evaluating Hemlibra® (emicizumab) prophylaxis administered every week or every two weeks in people with haemophilia A without factor VIII inhibitors and the phase III HAVEN 4 study evaluating Hemlibra prophylaxis administered every four weeks in people with haemophilia A with or without factor VIII inhibitors. 

"Hemlibra is the first medicine to show superior efficacy to prior factor VIII prophylaxis, the current standard of care therapy, as demonstrated by a statistically significant reduction in treated bleeds in the HAVEN 3 study intra-patient comparison," said Johnny Mahlangu, Faculty of Health Sciences, University of the Witwatersrand and NHLS, Johannesburg, South Africa. "Even with current prophylactic treatments, many people with haemophilia A continue to have bleeds that can lead to long-term joint damage, and there is a need for more treatment options."

In the phase III HAVEN 3 study, adults and adolescents aged 12 years or older without factor VIII inhibitors who received Hemlibra prophylaxis every week or every two weeks showed a 96% (p<0.0001) and 97% (p<0.0001) reduction in treated bleeds, respectively, compared to those who received no prophylaxis. In addition, 55.6% (95% CI: 38.1; 72.1) of people treated with Hemlibra every week and 60% (95% CI: 42.1; 76.1) of people treated with Hemlibra every two weeks experienced zero treated bleeds, compared to 0% (95% CI: 0.0; 18.5) of people treated with no prophylaxis. Importantly, in an intra-patient comparison, in patients who were previously enrolled in a prospective non-interventional study (NIS), once-weekly Hemlibra prophylaxis showed superior efficacy compared to prior factor VIII prophylaxis, the standard of care for people with haemophilia A without factor VIII inhibitors, as demonstrated by a 68% reduction (p<0.0001) in treated bleeds. Additionally, 93.7% (n=89/95; 95% CI, 86.8; 97.7) of all participants who completed a treatment preference survey preferred Hemlibra to their previous haemophilia treatment, with 97.8% (n=45/46) of those in the intra-patient comparison preferring Hemlibra to their prior factor VIII prophylaxis. 

"These new pivotal data show that Hemlibra controlled bleeds in people with haemophilia A, while offering the flexibility of less frequent subcutaneous dosing options," said Sandra Horning, MD, Roche’s chief medical officer and head of Global Product Development. "With this data, we now have positive results from all four of our phase III trials that reinforce the overall efficacy and safety of Hemlibra and its potential to improve care for all people with haemophilia A."

In the single-arm phase III HAVEN 4 study, adults and adolescents aged 12 years or older with or without factor VIII inhibitors receiving Hemlibra prophylaxis every four weeks had a median annualised bleeding rate for treated bleeds of 0.0 (IQR: 0.0; 2.1), with 56.1% (95% CI: 39.7; 71.5) of people experiencing zero treated bleeds and 90.2% (95% CI: 76.9; 97.3) experiencing three or fewer treated bleeds. Additionally, all participants (n=41/41; 95% CI, 91.4; 100.0) who responded to a patient preference survey preferred Hemlibra to their previous haemophilia treatment. 

In April 2018, the US Food and Drug Administration (FDA) granted Breakthrough Therapy Designation to Hemlibra for people with haemophilia A without factor VIII inhibitors, based on data from the HAVEN 3 study. Hemlibra was approved by the FDA in November 2017 for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adults and children with haemophilia A with factor VIII inhibitors based on results from the HAVEN 1 study and interim results from the HAVEN 2 study. Hemlibra was also recently approved by regulatory authorities in other countries around the world, including by the European Commission in February 2018 for routine prophylaxis of bleeding episodes in people with haemophilia A with factor VIII inhibitors.

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