Roche today announced new data for Hemlibra (emicizumab) across multiple pivotal studies in people with haemophilia A with and without factor VIII inhibitors at the International Society on Thrombosis and Haemostasis (ISTH) 2019 Congress on 6-10 July in Melbourne, Australia. In total, Roche presented 21 abstracts from its haemophilia programme, including five oral presentations. Further data from the four pivotal HAVEN clinical trials were presented, demonstrating the long-term safety, efficacy, and quality of life benefit of Hemlibra in people with haemophilia A with and without factor VIII inhibitors. Roche also presented the first interim analysis from the phase IIIb STASEY study, reinforcing the safety profile of Hemlibra in adults and adolescents (aged 12 years or older) with haemophilia A with factor VIII inhibitors seen in the HAVEN 1 clinical trial.
“Data presented at ISTH continues to reinforce Hemlibra’s potential to redefine the standard of care for people living with haemophilia A,” said Sandra Horning, MD, Roche’s Chief Medical Officer and Head of Global Product Development. “We are particularly excited to present the first interim analysis of safety data from the STASEY study, which adds to the growing body of evidence supporting Hemlibra as an important treatment option for people with haemophilia A.”
Updated data from the pooled HAVEN studies (HAVEN 1, HAVEN 2, HAVEN 3 and HAVEN 4; n=400), in people with haemophilia A of all ages with and without factor VIII inhibitors, showed that a high proportion of patients experienced zero treated bleeds on Hemlibra, and that this was maintained over a median of 83 weeks. Across all four HAVEN studies, over 87 percent of participants had no treated joint bleeds (either spontaneous or due to injury/trauma) and over 92 percent of participants experienced no spontaneous bleeds in each interval from week 25. Hemlibra’s established safety and tolerability profile was maintained.
Additionally, updated data from the HAVEN 3 and HAVEN 4 studies demonstrate that Hemlibra prophylaxis offers a clinically meaningful improvement in long-term health-related quality of life, versus previous episodic or prophylactic factor VIII treatment, for people with haemophilia A with and without factor VIII inhibitors as measured by the Haem-A-QoL questionnaire. In the 28 days prior to starting treatment with Hemlibra, 76 percent and 79 percent of employed patients from HAVEN 3 and HAVEN 4 studies, respectively, reported no missed days of work. At week 25 of HAVEN 3 and HAVEN 4, 91 percent and 93 percent of participants reported no missed workdays, respectively, with these figures remaining stable thereafter.
Results from the first interim analysis of the phase IIIb STASEY study, including data from 88 patients, reinforce the safety profile of Hemlibra characterised in the pivotal HAVEN 1 study. HAVEN 1 has formed the basis of Hemlibra’s approval in people with haemophilia A with factor VIII inhibitors in over 70 countries worldwide to date. In the STASEY study, in people with haemophilia A with factor VIII inhibitors, no cases of thrombotic microangiopathy or thrombotic events were reported and no new safety signals were identified. Eighteen (20.5 percent) patients reported a Hemlibra-related adverse event (AE), of which one was a serious AE (catheter site abscess). The most common AEs, occurring in 10 percent or more of people in the STASEY study were injection site reactions (14.8 percent), joint pain (arthralgia; 13.6 percent), headache (11.4 percent) and common cold symptoms (nasopharyngitis; 11.4 percent). Bleeding rates in people with haemophilia A with factor VIII inhibitors receiving Hemlibra in the STASEY study were also in line with previously reported observations from the HAVEN 1 study.
A retrospective analysis of pooled data across the HAVEN studies indicates that people with haemophilia A with and without factor VIII inhibitors had a reduced need for preventative (prophylactic) coagulation factor (factor VIII replacement therapy or bypassing agents) when undergoing certain minor surgeries. The majority of minor surgeries (n=215) were performed without prophylactic coagulation factor (n=141; 65.6 percent), and of these, 90.8 percet did not result in a treated post-operative bleed. Of the 18 major surgeries, three were managed without prophylactic coagulation factor, with no post-operative bleeds. The remaining 15 major surgeries were managed with prophylactic coagulation factor, only one of which resulted in a treated post-operative bleed.
Hemlibra is approved for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in people with haemophilia A with factor VIII inhibitors in over 70 countries worldwide, based on the results of the pivotal HAVEN 1 and HAVEN 2 studies. This includes the US in November 2017, EU member states in February 2018 and Japan in March 2018. Hemlibra has also been approved in people with haemophilia A without factor VIII inhibitors in over 40 countries worldwide, based on results from the pivotal HAVEN 3 and HAVEN 4 studies. This includes the US in October 2018, EU member states in March 2019 and Japan in December 2018. Submissions to other regulatory authorities around the world are ongoing.
