Study: Hemlibra Superior in Efficacy to Prior Factor VIII Prophylaxis in Hemophilia A

In a pivotal phase 3 study, hemophilia A treatment emicizumab-kxwh (Hemlibra) reduced bleeds in patients by 96% without factor VIII inhibitors and 68% compared with prior therapy, according to a press release.

The results are from Genentech’s two phase 3 studies: HAVEN 3 and HAVEN 4. The HAVEN 3 study, which included 152 participants, evaluated emicizumab-kxwh prophlyaxis administered every week or every 2 weeks in patients with hemophilia A without factor VIII inhibitors. In the HAVEN 4 study, emicizumab-kxwh was administered every 4 weeks.

In the HAVEN 3 study, adults and adolescents aged 12 years or older without factor VIII inhibitors administered emicizumab-kxwh prophylaxis every week or every 2 weeks showed a 96% and 97% reduction in treated bleads, respectively, compared with those who received no prophylaxis.

Other findings of the study include:

• 55.6% of patients treated with emicizumab-kxwh every week and 60% of patients treated with emicizumab-kxwh every 2 weeks experienced 0 bleeds.
• 93.7% of all patients who completed a treatment preference survey preferred emicizumab-kxwh to their previous hemophilia treatment.

In an intra-patient comparison, among patients who were previously enrolled in a prospective non-interventional study, once-weekly emicizumab-kxwh prophylaxis showed superior efficacy compared with prior factor VIII prophylaxis as demonstrated by a 68% reduction in treated bleeds, according to the study.

Based on data, the FDA granted breakthrough therapy designation to emicizumab-kxwh for patients with hemophilia A without factor VIII inhibitors.

The HAVEN 4 study included 48 patients with hemophilia A with or without factor VIII inhibitors who were previously treated with either factor VIII or bypassing agents, on demand or as prophylaxis.

According to the findings:

• 56.1% of patients experienced 0 treated bleeds and 90.2% experienced 3 or fewer treated bleeds.
• All participants preferred emicizumab-kxwh to their previous treatment.

The data demonstrate that emicizumab-kxwh administration every 4 weeks can provide clinically meaningful control of bleeding of patients with hemophilia A with or without factor VIII inhibitors, according to the press release.

“These new pivotal data show Hemlibra controlled bleeds in people with hemophilia A, while offering the flexibility of less frequent subcutaneous dosing options,” Sandra Horning, MD, Genentech chief medical officer and head of Global Product Development, said in the press release. “With this data, we now have positive results from all 4 of our phase 3 trials that reinforce the overall efficacy and safety of Hemlibra and its potential to improve care for all people with hemophilia A.”

The data were presented at the World Federation of Hemophilia 2018 World Congress in Glasgow, Scotland.

Hemlibra was created by Chugai Pharmaceutical Co and is being co-developed by Chugai, Roche, and Genentech.

Reference

Genentech’s HEMLIBRA (emicizumab-kxwh) Reduced Treated Bleeds by 96 Percent Compared to No Prophylaxis in Phase III HAVEN 3 Study in Hemophilia A without Factor VIII Inhibitors [news release]. Genentech’s website. https://www.gene.com/media/press-releases/14723/2018-05-20/genentechs-hemlibra-emicizumab-kxwh-redu. Accessed May 21, 2018.