- Phase III HAVEN 7 primary data presented at ASH 2023 provide additional confidence in the favourable efficacy and safety profile of subcutaneous Hemlibra given soon after birth 1
- At nearly two years median follow-up in the descriptive, single-arm study, no babies experienced spontaneous bleeds requiring treatment, and all treated bleeds were as a result of trauma 1
- Safety results were consistent with previous studies of Hemlibra, with no new safety signals observed 1
- The HAVEN 7 study was developed in collaboration with the haemophilia A community, to generate additional evidence for the prophylactic treatment of infants with haemophilia A
“Haemophilia A can have a devastating impact on any patient, but this is especially true for infants, where the emotional and physical stress due to frequent hospital visits, treatment administration and other worries can be distressing for babies and their parents and caregivers,” said
The burden of severe haemophilia A in babies and on their parents and caregivers is significant.
“The results of HAVEN 7 provide additional confidence in the efficacy and safety profile of Hemlibra for babies with severe haemophilia A, and add to its extensive clinical and real-world evidence across all ages,” said
The HAVEN 7 study is a Phase III, descriptive, single-arm study, set up in collaboration with the haemophilia A community to evaluate the efficacy, safety, pharmacokinetics and pharmacodynamics of subcutaneous Hemlibra in infants with severe haemophilia A without factor VIII inhibitors. These results, which included data from 55 participants, showed that at 101.9 weeks median follow-up, 54.5% of participants (n=30) did not have any bleeds that required treatment, while 16.4% (n=9) did not have any treated or untreated bleeds at all. There were no spontaneous bleeds requiring treatment in any participant, and all treated bleeds were as a result of trauma. A total of 207 bleeds occurred in 46 participants (83.6%); 87.9% of these were as a result of trauma. Model-based annualised bleeding rate (95% CI) was 0.4 (0.30–0.63) for treated bleeds. No new safety signals were observed and there were no treatment-related serious adverse events, intracranial haemorrhages or deaths reported. Just 3.6% of participants (n=2) tested positive for factor VIII inhibitors, which may be a consequence of reduced factor VIII usage in participants treated with Hemlibra, and no participant tested positive for anti-drug antibodies.[1] Results were consistent with positive results from the interim analysis and from previous Phase III HAVEN studies.10-14
The results of additional research on biomarkers in the HAVEN 7 study were also presented at ASH, and were supportive of the study’s primary efficacy analysis. This additional research showed that the pharmacodynamic profiles of Hemlibra in babies were consistent with those previously observed in older children and adults with haemophilia A. The data showed that Hemlibra exhibits the expected pharmacodynamic response, despite the reduced presence of the clotting factors that Hemlibra binds to in this age group.15
The Phase III HAVEN 7 study results complement data from the broader, pivotal HAVEN clinical programme, providing insights into the evolution of haemophilia A in babies, and the impact of initiating preventative treatment from birth. The primary analysis is being followed by a seven year extension period.1 Hemlibra continues to redefine standards of care in haemophilia A, as a flexible treatment option approved across all ages and stages of life, regardless of inhibitor status and at different dosing frequencies. It is approved for the routine prophylaxis of people with haemophilia A in more than 115 countries worldwide. It has been studied in one of the largest clinical trial programmes in people with haemophilia A with and without factor VIII inhibitors, including eight Phase III studies.
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).9 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.2,16 The burden of severe haemophilia A in infants and on their parents and caregivers is significant. 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 symptoms, people with haemophilia A can bleed frequently, especially into their joints or muscles.17 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.18 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.2
About
About
Founded in 1896 in
In recognising our endeavour to pursue a long-term perspective in all we do,
For more information, please visit www.roche.com.
All trademarks used or mentioned in this release are protected by law.
References
[1] Pipe S, et al. Emicizumab Prophylaxis for the Treatment of Infants with Severe Hemophilia A without Factor VIII Inhibitors: Results from the Primary Analysis of the HAVEN 7 Study. Presented at
[2] Srivastava A, et al. WFH guidelines for the management of hemophilia, 3rd edition. Haemophilia. 2020;26 (Suppl 6): 1-158.
[3] Manco-Johnson MJ, et al. Prophylaxis versus episodic treatment to prevent joint disease in boys with severe hemophilia.
[4] Andersson NG, et al. Intracranial haemorrhage in children and adolescents with severe haemophilia A or B – the impact of prophylactic treatment.
[5] Spagrud LJ, et al. Pain, distress, and adult-child interaction during venipuncture in pediatric oncology: an examination of three types of venous access.
[6] Van den Berg HM, et al. Timing of inhibitor development in more than 1000 previously untreated patients with severe hemophilia A. Blood 2019;134:317–320.
[7] Valentino LA & Kapoor M. Central venous access devices in patients with hemophilia. Expert Rev Med Devices 2005;2:699–711.
[8] Mancuso M, et al. Prophylaxis in children with haemophilia in an evolving treatment landscape. Haemophilia. 2021;27:889–896.
[9] Hemlibra SmPC [Internet; cited 2023 December] Available from: https://www.medicines.org.uk/emc/product/9043/smpc
[10] Pipe S, et al. Emicizumab Prophylaxis for the Treatment of Infants with Severe Hemophilia A without Factor VIII Inhibitors: Results from the Interim Analysis of the HAVEN 7 Study. Presented at
[11] 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.
[12] 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.
[13] Mahlangu J, et al. Emicizumab Prophylaxis in Patients Who Have Hemophilia A without Inhibitors. N Engl J Med. 2018;379:811-822.
[14] Pipe S, et al. Efficacy, safety, and pharmacokinetics of emicizumab prophylaxis given every 4 weeks in people with haemophilia A (HAVEN 4): a multicentre, open-label, non-randomised phase 3 study. The Lancet Haematology. 2019; DOI:https://doi.org/10.1016/S2352-3026(19)30054-7.
[15] Pipe S, et al. Pharmacodynamic Biomarkers in Infants with Hemophilia A Receiving Emicizumab in HAVEN 7. Presented at
[16] Iorio A, et al. Establishing the Prevalence and Prevalence at Birth of Hemophilia in Males. Ann Intern Med. 2019;171(8):540-546.
[17] NHS. Symptoms of haemophilia [Internet; cited 2023 December]. Available from: https://www.nhs.uk/conditions/haemophilia/symptoms/.
[18] Franchini M, et al. Haemophilia A in the third millennium. Blood Rev. 2013; 179-84.
Roche Global Media Relations
Phone: +41 61 688 8888 / e-mail: media.relations@roche.com
Hans Trees, PhD Phone: +41 79 407 72 58 | Dr. Phone: +41 79 205 27 03 |
Phone: +44 797 32 72 915 | Phone: +41 79 461 86 83 |
Nina Mählitz Phone: +41 79 327 54 74 | Phone: +49 172 6367262 |
Sileia Urech Phone: +41 79 935 81 48 |
Roche Investor Relations
Dr. Phone: +41 61 68-75284 e-mail: bruno.eschli@roche.com | Dr. Phone: +41 61 68-88027 e-mail: sabine.borngraeber@roche.com |
Dr. Birgit Masjost Phone: +41 61 68-84814 e-mail: birgit.masjost@roche.com | Dr. Phone: +41 61 68-72942 e-mail: gerard.tobin@roche.com |
Investor Relations
Phone: +1 650 225 3217 e-mail: kalm.loren@gene.com |
Attachment
- 09122023_MR_ASH Hemlibra HAVEN 7_en
© OMX, source