LIFE FIRST, HAEMOPHILIA A SECOND

HEMLIBRA® is the first and only bispecific1,2 that
revolutionised haemophilia A care,3–5 with over a
decade of proven evidence6,7,22 and transformational
experience8,9 in over 30,000 patients worldwide10

Product photo

MORE THAN A DECADE OF:
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BLEED PROTECTION & BEYOND

HEMLIBRA® is the only bispecific for Patients with Haemophilia A (PwHA)<sup>1,2</sup>

included in the WHO list of essential medicines.<sup>3</sup> With over a decade of bleed protection data and beyond,<sup>6,7,22</sup> ~80% of patients achieved zero treated bleeds,<sup>7,16,17,25,26</sup> 94% maintained or improved their clinical joint health<sup>11</sup> and up to 75% adopted a more active lifestyle after switching.<sup>12</sup>
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n = number of patients who contributed data for analyses at each time interval. Group sizes differ because patients at any point could choose to convert to commercial drug or leave the study for any reason, which excludes them for further analysis in the study. By May 2020, many had switched to commercially available HEMLIBRA® or stopped treatment for other reasons.7

These data are a descriptive analysis and therefore should be interpreted with caution.

Data from 401 paediatric and adult PwHA with or without FVIII inhibitors who were enrolled in the Phase III HAVEN 1, HAVEN 2, HAVEN 3 and HAVEN 4 studies, pooled to establish a long-term efficacy, safety and pharmacokinetics profile.7
Icon representing proven efficacy

MAINTAINED OR IMPROVED THEIR CLINICAL JOINT HEALTH<sup>11</sup>

In the Phase IV, multicentre BEYOND ABR study (N=130), in patients with moderate/severe haemophilia A who had previously received at least 24 weeks of factor VIII prophylaxis, improvement in HJHS was observed from baseline to Month 12 after switching from FVIII prophylaxis to HEMLIBRA®.11
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ADOPTED A MORE ACTIVE LIFESTYLE AFTER SWITCHING<sup>12</sup>

Recent data from the cross-sectional, non-interventional HEMO-ACT study in 56 males with severe haemophila A showed positive impacts on enhancing physical activity after patients switched to HEMLIBRA®.12

66.1%

reported increased physical activity<sup>12</sup>

73.2%

perceived a reduced bleeding risk<sup>12</sup>

78.6%

felt safer during physical activity<sup>12</sup>
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EASE OF ADMINISTRATION

HEMLIBRA® is the only approved subcutaneous bispecific for PwHA.<sup>1,2</sup>

It offers the flexibility of weekly, every-2-week or every-4-week dosing options,<sup>1</sup> and is preferred by up to 96% of patients versus their prior therapy.<sup>11,23,24</sup>

Two-thirds of patients are treated every 2 weeks or every 4 weeks, offering flexible dosing options to fit into their lifestyles.<sup>1,13</sup>
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OF PATIENTS PREFERRED HEMLIBRA® TO THEIR PRIOR THERAPY<sup>11,23,24</sup>
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SAFETY

HEMLIBRA® is the only bispecific in haemophilia A with over a decade of clinical experience, and with an established safety profile,<sup>1,2,6,7,14,22</sup> including use in infants.<sup>1,15</sup>
  • See full prescribing information for warnings and precautions, adverse reactions and drug interactions.1
  • Thrombotic microangiopathy and thromboembolism: Cases of thrombotic microangiopathy and thrombotic events were reported when on average a cumulative amount of >100 U/kg/24 hours of activated prothrombin complex concentrate (aPCC) was administered for 24 hours or more to patients receiving HEMLIBRA® prophylaxis. Monitor for the development of thrombotic microangiopathy and thrombotic events if aPCC is administered. Discontinue aPCC and suspend dosing of HEMLIBRA® if symptoms occur.1,14
  • Immunogenicity: Anti-emicizumab antibodies (including neutralising antibodies) have developed in HEMLIBRA®-treated patients. In case of clinical signs of loss of efficacy, promptly assess the aetiology and consider a change in treatment if neutralizing antibodies are suspected.1
  • Laboratory coagulation test interference: HEMLIBRA® interferes with activated clotting time (ACT), activated partial thromboplastin time (aPTT), and coagulation laboratory tests based on aPTT, including one-stage aPTT-based single-factor assays, aPTT-based Activated Protein C Resistance (APC-R), and Bethesda assays (clotting-based) for FVIII inhibitor titres. Intrinsic pathway clotting-based laboratory tests should not be used.1

Long-term safety profile of the HAVEN 1–4 studies<sup>7</sup>:

• No new safety concerns were identified in the long-term descriptive portion of the clinical studies (N=399 over a median period of 130 weeks)

In the pooled Phase III studies, all ISRs were non-serious (mild or moderate) in intensity, and 94.9% resolved without treatment<sup>1</sup>

AEs reported in ≥10% of patients¹

n(%)

Injection site reaction

19.4%

Arthralgia

14.2%

Headache

14.0%

HAVEN 7 : Safety in infants

HAVEN 7 safety profile:

Primary analysis confirmed HEMLIBRA® was well tolerated in infants with severe HA without FVIII inhibitors, including PUPs (N=55)<sup>15</sup>. No intracranial hemorrhages occurred. There were no treatment-related SAEs, and no AEs led to withdrawals, dose modifications, or interruptions.

IMPORTANT SAFETY INFORMATION

 

 

INDICATION

HEMLIBRA is indicated for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients ages newborn and older with hemophilia A with or without factor VIII inhibitors.

 

IMPORTANT SAFETY INFORMATION Boxed WARNING: THROMBOTIC MICROANGIOPATHY and THROMBOEMBOLISM Cases of thrombotic microangiopathy and thrombotic events were reported when on average a cumulative amount of >100 U/kg/24 hours of activated prothrombin complex concentrate (aPCC) was administered for 24 hours or more to patients receiving HEMLIBRA prophylaxis. Monitor for the development of thrombotic microangiopathy and thrombotic events if aPCC is administered. Discontinue aPCC and suspend dosing of HEMLIBRA if symptoms occur.

Warnings and Precautions
Thrombotic Microangiopathy (TMA) and Thromboembolism Associated With HEMLIBRA and aPCC

In clinical trials, TMA was reported in 0.8% of patients (3/391) and thrombotic events were reported in 0.5% of patients (2/391). In patients who received at least one dose of aPCC, TMA was reported in 8.1% of patients (3/37) and thrombotic events were reported in 5.4% of patients (2/37). Patients with TMA presented with thrombocytopenia, microangiopathic hemolytic anemia, and acute kidney injury, without severe deficiencies in ADAMTS13.

Consider the benefits and risks if aPCC must be used in a patient receiving HEMLIBRA prophylaxis. Due to the long half-life of HEMLIBRA, the potential for an interaction with aPCC may persist for up to 6 months after the last dose. Monitor for the development of TMA and/or thromboembolism when administering aPCC. Immediately discontinue aPCC and interrupt HEMLIBRA prophylaxis if clinical symptoms, imaging, or laboratory findings consistent with TMA and/or thromboembolism occur, and manage as clinically indicated. Consider the benefits and risks of resuming HEMLIBRA prophylaxis following complete resolution of TMA and/or thrombotic events on a case-by-case basis.

Immunogenicity
Treatment with HEMLIBRA may induce anti-drug antibodies. Anti-emicizumab-kxwh antibodies were reported in 5.1% of patients (34/668) treated with HEMLIBRA in clinical trials. Most patients with anti-emicizumab-kxwh antibodies did not experience a change in HEMLIBRA plasma concentrations or an increase in bleeding events; however, in uncommon cases (incidence <1%), the presence of neutralizing antibodies with decreasing plasma concentration may be associated with loss of efficacy.
Monitor for clinical signs of loss of efficacy (eg, increase in breakthrough bleeding events) and if observed, promptly assess the etiology and consider a change in treatment if neutralizing anti-emicizumab kxwh antibodies are suspected.

Laboratory Coagulation Test Interference
HEMLIBRA affects intrinsic pathway clotting-based laboratory tests, including activated clotting time (ACT); activated partial thromboplastin time (aPTT); and all assays based on aPTT, such as one-stage, factor VIII (FVIII) activity. Therefore, intrinsic pathway clotting-based coagulation laboratory test results in patients who have been treated with HEMLIBRA prophylaxis should not be used to monitor HEMLIBRA activity, determine dosing for factor replacement or anti-coagulation, or measure FVIII inhibitor titers. Results affected by HEMLIBRA: aPTT; Bethesda assays (clotting-based) for FVIII inhibitor titers; one stage, aPTT-based single-factor assays; aPTT-based Activated Protein C Resistance (APC-R); ACT. Results unaffected by HEMLIBRA: Bethesda assays (bovine chromogenic) for FVIII inhibitor titers; thrombin time (TT); one-stage, prothrombin time (PT)-based single-factor assays; chromogenic-based single-factor assays other than FVIII (see Drug Interactions for FVIII chromogenic activity assay considerations); immuno-based assays (ie, ELISA, turbidimetric methods); genetic tests of coagulation factors (eg, Factor V Leiden, Prothrombin 20210).

Most Common Adverse Reactions
The most common adverse reactions (incidence ≥10%) are injection site reactions, headache, and arthralgia

Adverse Reactions
Characterization of aPCC Treatment in Pooled Clinical Trials
There were 130 instances of aPCC treatment in 37 patients, of which 13 instances (10%) consisted of on average a cumulative amount of >100 U/kg/24 hours of aPCC for 24 hours or more; 2 of the 13 were associated with thrombotic events and 3 of the 13 were associated with TMA. No TMA or thrombotic events were associated with the remaining instances of aPCC treatment.

Injection Site Reactions
In total, 85 patients (22%) reported injection site reactions (ISRs). All ISRs observed in HEMLIBRA clinical trials were reported as mild to moderate intensity and 93% resolved without treatment. The commonly reported ISR symptoms were injection site erythema (11%), injection site pruritus (4%), and injection site pain (4%).

Other Less Common (<1%) Reactions
Rhabdomyolysis was reported in 2 adult patients with asymptomatic elevations in serum creatine kinase without associated renal or musculoskeletal symptoms. In both instances, the event occurred following an increase in physical activity.

Drug Interactions
Clinical experience suggests that a drug interaction exists with HEMLIBRA and aPCC.

Pregnancy, Lactation, Females and Males of Reproductive Potential
Women of childbearing potential should use contraception while receiving HEMLIBRA. It is not known whether HEMLIBRA can cause fetal harm when administered to a pregnant woman or can affect reproduction capacity. HEMLIBRA should be used during pregnancy only if the potential benefit for the mother outweighs the risk to the fetus. The developmental and health benefits of breastfeeding should be considered along with the mother’s clinical need for HEMLIBRA and any potential adverse effects on the breastfed child from HEMLIBRA or from the underlying maternal condition.

Please see accompanying HEMLIBRA full Prescribing Information for additional Important Safety Information.
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TRUST

HEMLIBRA® is the only bispecific for haemophilia A with over a decade of clinical experience,<sup>1,2,6,7,22</sup> trusted by >30,000 patients worldwide.<sup>10</sup>
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HAEMOPHILIA FREE MIND

Discover the Haemophilia-Free Mind concept

Learn more about how HEMLIBRA® can help patients to live with a “haemophilia-free mind”<sup>21</sup>
References
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  2. Mannucci PM. Hemophilia therapy: the future has begun. Haematologica. 2020;105(3):545–53.
  3. World Health Organization (WHO). Model List of Essential Medicines – 24th List, 2025.
  4. Alves de Jesus VE, Lamounier de Almeida D, Júnior RLM, et al. Factor-VIII mimetic bispecific antibodies for the treatment of hemophilia A: an update. Expert Rev Clin Pharmacol 2026; Epub ahead of print.
  5. Srivastava A, Santagostino E, Dougall A, et al. WFH Guidelines for the Management of Hemophilia, 3rd edition. Haemophilia. 2020;26:1–158.
  6. Shima M, Nagao A, Taki M, et al. Long-term safety and efficacy of emicizumab for up to 5.8 years and patients’ perceptions of symptoms and daily life: A phase 1/2 study in patients with severe haemophilia A. Haemophilia. 2021;27(1):81–9.
  7. Callaghan MU, Negrier C, Paz-Priel I, et al. Long-term outcomes with emicizumab prophylaxis for hemophilia A with or without FVIII inhibitors from the HAVEN 1-4 studies. Blood. 2021;137(16):2231–42.
  8. Araujo IO, Suassuna LF, dos Santos IL, et al. Efficacy, safety and satisfaction of using emicizumab in hemophilia A patients without factor VIII inhibitors: A systematic review. Hematol Transfus Cell Ther. 2025;47(3):103849.
  9. Alcedo Andrade PE, Manucci PM & Kessler CM. Emicizumab: the hemophilia A game-changer. Haematologica. 2024;109(5):1334–1347.
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  11. Kruse-Jarres R, Castaman G, Minno M, et al. Evolution of joint health and physical activity in people with hemophilia A without factor VIII inhibitors switching to emicizumab prophylaxis: A second interim analysis of the BEYOND ABR study. Presented at: 67th American Society of Hematology (ASH) Annual Meeting; 2025 Dec 6–9. Orlando, FL, USA. Poster #P1285.
  12. De la Corte H, Carrasco Expósito M, Ríos de Paz M, et al. Enhancing Physical Activity in Patients Treated with Emicizumab in Hemophilia A: Results from the HEMO-ACT Cross-Sectional Study. Presented at: European Association for Haemophilia and Allied Disorders Congress; 2026 Feb 3–6. Dublin, Ireland. Abstract #PO073.
  13. Roche data on file.
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  18. World Federation of Hemophilia. Annual Global Survey Report 2024. Available at: https://www1.wfh.org/publications/files/pdf-2588.pdf (Accessed March 18, 2026).
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