New Data Reinforce Long-Term Benefit of Emicizumab for Patients With Hemophilia A


The data presented at ASH 2020 build on results previously observed in the pivotal HAVEN clinical trial among adults, adolescents, and children with hemophilia A, with and without factor VIII inhibitors.

Results from a new analysis reinforce the long-term efficacy and safety profile of emicizumab (Hemlibra; Roche and Genentech), according to a presentation by Roche at the virtual 62nd American Society of Hematology (ASH) Annual Meeting and Exposition.1 The analysis pooled the 3-year follow-up data of 401 people—adults, adolescents, and children—with hemophilia A with and without factor VIII (FVIII) inhibitors from the pivotal HAVEN 1-4 studies.

Hemophilia A is a serious, inherited condition in which a person’s blood does not clot properly, leading to uncontrolled and often spontaneous bleeding. According to Roche, the disorder affects approximately 320,000 people worldwide.

“The long-term benefit of [emicizumab], 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, Roche’s chief medical officer and head of Global Product Development, in a prepared statement. “As the first new class of medicine in nearly 20 years, [emicizumab] 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.”

The latest analysis showed the 4 HAVEN studies (HAVEN 1, n=113; HAVEN 2, n=88; HAVEN 3, n=152; HAVEN 4, n=48) had a median duration efficacy period of 120.4 weeks. Emicizumab, a bispecific factor IXa- and factor X-directed antibody, maintained low treated bleed rates across the study period, with model based annualized bleed rates (ABR) remaining low throughout the evaluation period at 1.4 (95% CI: 1.1-1.7). Further, the proportion of participants who experienced 0 treated bleeds (70.8-83.7%) increased with each consecutive 24-week period.

Additionally, with emicizumab prophylaxis, 95.1% of target joints were resolved. Results also showed that emicizumab’s safety profile was consistent with previous observations and no new safety signals were observed after the longer follow-up.

Furthermore, the first interim analysis of the European Haemophilia Safety Surveillance (EUHASS) Database, was also presented at the ASH meeting, suggests the safety profile of emicizumab in the real-world setting is consistent with that seen in clinical trials, with no new or emerging safety signals.

According to Roche, data from the HAVEN studies supported emicizumab approvals to treat people with hemophilia A with FVIII inhibitors in more than 90 countries and for people without FVIII inhibitors in more than 80 countries, including the United States. Since its initial approval more than 3 years ago, 8200 people have received emicizumab globally, and in the United States, the drug is now the most prescribed prophylactic treatment for hemophilia A.


  • New follow-up phase III data reinforce the long-term benefit of Roche’s Hemlibra for people with haemophilia A [news release]. Basel, Switzerland; December 7, 2020: Roche. Accessed December 10, 2020.

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