Study: Emicizumab Demonstrates Long-Term Benefit for Patients with Hemophilia A

New follow-up phase 3 trial data show that with nearly 3 years of follow-up, emicizumab (Hemlibra, Roche) maintained low treated bleed rates and was well tolerated in patients of all ages with hemophilia A, both with and without factor VIII (FVIII) inhibitors.

Hemophilia A is an inherited disorder that causes an individual’s blood to not clot properly, leading to uncontrolled and spontaneous bleeding. It affects approximately 320,000 people worldwide, according to a press release, and between 50% and 60% of these patients have a severe form of the disorder.

Patients with hemophilia A either have very little or no FVIII, a clotting factor that helps form clots and stop bleeding in healthy individuals. Although many patients with hemophilia A receive FVIII as part of their treatment, the development of inhibitors to FVIII replacement therapies can be a major complication. According to the press release, inhibitors are antibodies developed by the body’s immune system that bind to and block the replacement FVIII, limiting its ability to control bleeding.

The new results are from pooled, 3-year follow-up data of 401 patients with hemophilia A from the HAVEN studies 1 through 4. The data were presented at the virtual 62^nd American Society of Hematology (ASH) Annual Meeting and Exposition.

The HAVEN studies had a median duration efficacy period of 120.4 weeks, during which time emicizumab maintained low treated bleed rates. Model-based annualized bleed rates also remained low throughout the study period. Furthermore, the proportion of patients who experienced zero treated bleeds increased with each consecutive 24-week period, according to the press release. The trial results also found that the safety profile was consistent with previous observations and no new safety signals were observed after the longer follow-up.

“The long-term benefit of Hemlibra, with a consistent safety profile and durably effective control of bleeding, underscores its potential to redefine the standard of care for people living with hemophilia A,” said Levi Garraway, MD, PhD, chief medical officer at Roche, in a press release. “As the first new class of medicine in nearly 20 years, Hemlibra continues to show a positive impact on disease burden and quality of life for people with hemophilia A, regardless of their inhibitor status or age.”

In other data presented at the ASH Meeting and Exposition, the first interim analysis of the European Haemophilia Safety Surveillance Database found that the safety profile of emicizumab in the real-world setting is consistent with that seen in clinical trials. The database is a large pharmacovigilance program monitoring the safety of treatments for inherited bleeding disorders.

REFERENCE

New follow-up phase III data reinforce the long-term benefit of Roche’s Hemlibra for people with haemophilia A [news release]. Roche; December 7, 2020. https://www.roche.com/media/releases/med-cor-2020-12-07.htm. Accessed December 7, 2020.