ROCHE HOLDING AG * People with moderate or mild haemophilia A have significant unmet clinical needs, as this population may not use preventative treatments due to missed or delayed diagnoses of bleeding episodes and a lack of treatment guidelines1,2
* New data indicate that Hemlibra has a favourable safety profile in people with moderate or mild haemophilia A without factor VIII inhibitors, with no new safety signals identified3
* Hemlibra also achieved clinically meaningful bleed control, with 80.3% of participants experiencing no bleeding episodes that required treatment and 90.1% experiencing no joint bleeds that required treatment3
* A separate analysis of thrombosis-related events in people taking Hemlibra, including real-world data, further confirmed the safety profile of Hemlibra4
While the treatment and management of severe haemophilia A are well-established, there is less information and treatment guidance on moderate and mild haemophilia A, which can lead to delayed or missed diagnosis of bleeding episodes.1 Considering this population may not use preventative treatments, they may experience worsened clinical burden, with less than 30% of people with moderate or mild haemophilia A living a bleed-free life.1,2
"We are pleased to see that Hemlibra continues to show benefit in additional haemophilia A populations, regardless of severity," said
HAVEN 6 is a phase III study evaluating the safety, efficacy, pharmacokinetics and pharmacodynamics of Hemlibra in people with moderate or mild haemophilia A without factor VIII inhibitors. This interim analysis included data from 71 participants (69 men and two women); 20 of whom had mild haemophilia A without factor VIII inhibitors and 51 had moderate haemophilia A without inhibitors. Thirty-seven participants were on factor VIII prophylaxis at baseline.3
This interim analysis was conducted after 50 participants with moderate haemophilia A completed at least 24 weeks in the study or withdrew. Data cut-off was on
The most common adverse events (AEs) occurring in 10% or more people in the HAVEN 6 study were headache (14.1%) and local injection site reactions (ISRs) (12.7%). Eleven people (15.5%) reported a Hemlibra-related AE, with ISRs being the most common (12.7%). There were no deaths, or cases of thrombotic microangiopathy (TMA) or serious thrombotic events (TEs) in the study as of the data cut-off, reinforcing Hemlibra's favourable safety profile.3
A separate analysis of TE and TMA events in people taking Hemlibra, including real-world data, will also be presented as a poster at ASH.4 These results showed that the evaluation of reported events without concomitant activated prothrombin complex concentrate (aPCC) remains similar to previous analyses as exposure increases, and the benefit/risk profile of Hemlibra remains unchanged. These data further confirm the favourable safety profile of Hemlibra, consistent with results from previous HAVEN and STASEY studies.4,10
Hemlibra is approved to treat people with haemophilia A with factor VIII inhibitors in more than 100 countries worldwide and for people without factor VIII inhibitors in more than 90 countries worldwide, including the US, EU and
About Hemlibra® (emicizumab)
Hemlibra is a bispecific factor IXa- and factor X-directed antibody. It is designed to bring together factor IXa and factor X, proteins involved in the natural coagulation cascade, and restore the blood clotting process for people with haemophilia A. Hemlibra is a prophylactic (preventative) treatment that can be administered by an injection of a ready-to-use solution under the skin (subcutaneously) once-weekly, every two weeks, or every four weeks (after an initial once-weekly dose for the first four weeks). Hemlibra was created by
About haemophilia A
Haemophilia A is an inherited, serious disorder in which a person's blood does not clot properly, leading to uncontrolled and often spontaneous bleeding. Haemophilia A affects around 900,000 people worldwide, approximately 35-39% of whom have a severe form of the disorder. People with haemophilia A either lack or do not have enough of a clotting protein called factor VIII. In a healthy person, when a bleed occurs, factor VIII brings together the clotting factors IXa and X, which is a critical step in the formation of a blood clot to help stop bleeding. Depending on the severity of their disorder, people with haemophilia A can bleed frequently, especially into their joints or muscles. These bleeds can present a significant health concern as they often cause pain and can lead to chronic swelling, deformity, reduced mobility and long-term joint damage. A serious complication of treatment is the development of inhibitors to factor VIII replacement therapies. Inhibitors are antibodies developed by the body's immune system that bind to and block the efficacy of replacement factor VIII, making it difficult, if not impossible, to obtain a level of factor VIII sufficient to control bleeding.
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References
[1] Walsh C, et al. Identified unmet needs and proposed solutions in mild-to-moderate haemophilia: A summary of opinions from a roundtable of haemophilia experts. Haemophilia. 2021;27(S1):25-32.
[2] Nissen F, et al. An Insight into clinical outcomes in mild, moderate, and severe hemophilia A (HA): A preliminary analysis of the CHESS II study. Presented at:
[3] Negrier C, et al. Emicizumab Prophylaxis in Persons with Mild or Moderate Hemophilia A: Results from the Interim Analysis of the HAVEN 6 Study. Presented at:
[4] Howard M, et al. Evaluation of the Safety of Emicizumab Prophylaxis in Persons with Hemophilia A: An Updated Summary of Thrombotic Events and Thrombotic Microangiopathies. Presented at:
[5] Mancuso ME, et al. Emicizumab Prophylaxis in Adolescent/Adult Patients with Hemophilia A Previously Receiving Episodic or Prophylactic Bypassing Agent Treatment: Updated Analyses from the HAVEN 1 Study. Blood. 2017;130 (Supplement 1):1071.
[6] Young G, et al. Emicizumab prophylaxis provides flexible and effective bleed control in children with hemophilia A with inhibitors: results from the HAVEN 2 study. Blood. 2018;132 (Supplement 1):632.
[7] Mahlangu J, et al. Emicizumab Prophylaxis in Patients
[8] Pipe S, et al. Emicizumab subcutaneous dosing every 4 weeks is safe and efficacious in the control of bleeding in persons with hemophilia A (PwHA) with and without inhibitors: Results from the Phase 3 HAVEN 4 study [abstract no. M-LBMED01-005 (854)]. Haemophilia. 2018;24:209-218.
[9] Parnes A, et al. Patient preference for emicizumab versus prior factor therapy in people with haemophilia A: Results from the HAVEN 3 and HAVEN 4 studies. Haemophilia. 2021;27:e772-e775.
[10] Lee L, et al. Summary of thrombotic or thrombotic microangiopathy events in persons with hemophilia A taking Emicizumab. Presented at:
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