Emicizumab

HEMLIBRA bridges activated factor IX and factor X to restore the function of missing activated factor VIII that is needed for effective hemostasis.

• 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 (congenital factor VIII deficiency) with or without factor VIII inhibitors. HEMLIBRA is a bispecific factor IXa- and factor X-directed antibody 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 (congenital factor VIII deficiency) with or without factor VIII inhibitors.

• None.contraindicated

Recommended loading dose is 3 mg/kg by subcutaneous injection once weekly for the first 4 weeks, followed by a maintenance dose of: 1.5 mg/kg once every week, or 3 mg/kg once every two weeks, or 6 mg/kg once every four weeks. ( 2.1 ) See Full Prescribing Information for important preparation and administration instructions. ( 2.2 ) 2.1 Recommended Dosage For subcutaneous use only. The recommended loading dose is 3 mg/kg by subcutaneous injection once weekly for the first 4 weeks, followed by a maintenance dose of: 1.5 mg/kg once every week, or 3 mg/kg once every two weeks, or 6 mg/kg once every four weeks. The selection of a maintenance dose should be based on healthcare provider preference with consideration of regimens that may increase patient adherence. Discontinue the prophylactic use of bypassing agents the day before starting HEMLIBRA prophylaxis. The prophylactic use of factor VIII (FVIII) products may be continued during the first week of HEMLIBRA prophylaxis. Missed Dose If a dose of HEMLIBRA is missed administer as soon as possible and then resume usual dosing schedule. Do not administer two doses on the same day to make up for a missed dose. 2.2 Preparation and Administration HEMLIBRA is intended for use under the guidance of a healthcare provider. After proper training in subcutaneous injection technique, a patient may self-inject, or the patient's caregiver may administer HEMLIBRA, if a healthcare provider determines that it is appropriate. Self-administration is not recommended for children less than 7 years of age.

Immunogenicity: Anti-emicizumab antibodies (including neutralizing antibodies) have developed in HEMLIBRA-treated patients. In case of clinical signs of loss of efficacy, promptly assess the etiology and consider a change in treatment if neutralizing antibodies are suspected. ( 5.3 , 12.6 , 14.3 ) 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 factor VIII (FVIII) inhibitor titers. Intrinsic pathway clotting-based laboratory tests should not be used. ( 5.4 , 7.2 ) 5.1 Thrombotic Microangiopathy Associated with HEMLIBRA and aPCC Cases of thrombotic microangiopathy (TMA) were reported from clinical trials 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. In clinical trials, thrombotic microangiopathy was reported in 0.8% of patients (3/391) and in 8.1% of patients (3/37) who received at least one dose of aPCC. Patients presented with thrombocytopenia, microangiopathic hemolytic anemia, and acute kidney injury, without severe deficiencies in ADAMTS13 activity. Evidence of improvement was seen within one week following discontinuation of aPCC. One patient resumed HEMLIBRA following resolution of TMA.

The following serious adverse reactions are described elsewhere in the labeling: Thrombotic Microangiopathy Associated with HEMLIBRA and aPCC [see Warnings and Precautions (5.1) ] Thromboembolism Associated with HEMLIBRA and aPCC [see Warnings and Precautions (5.2) ] Most common adverse reactions (incidence ≥ 10%) are injection site reactions, headache, and arthralgia. ( 6.1 ) To report SUSPECTED ADVERSE REACTIONS, contact Genentech at 1-888-835-2555 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch. 6.1 Clinical Trials Experience Because clinical trials are conducted under widely varying conditions, adverse reaction rates observed in the clinical trials of a drug cannot be directly compared to rates in the clinical trials of another drug and may not reflect the rates observed in practice. The following adverse reactions are based on pooled data from two randomized trials in adult and adolescent patients (HAVEN 1 and HAVEN 3), one single-arm trial in adult and adolescent patients (HAVEN 4), one single-arm trial in pediatric patients (HAVEN 2), and one dose-finding trial, in which a total of 391 male patients with hemophilia A received at least one dose of HEMLIBRA as routine prophylaxis. Two hundred eighty-one patients (72%) were adults (18 years and older), 50 (13%) were adolescents (12 years up to less than 18 years), 55 (14%) were children (2 years up to less than 12 years), and five (1%) were infants (1 month up to less than 2 years). The median duration of exposure across the studies was 34.1 weeks (0.1 to 224.4 weeks).

8.1 Pregnancy Risk Summary There are no available data on HEMLIBRA use in pregnant women to inform a drug-associated risk of major birth defects and miscarriage. Animal reproduction studies have not been conducted with emicizumab-kxwh. 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. All pregnancies have a background risk of birth defect, loss, or other adverse outcomes. The estimated background risk of major birth defects and miscarriage for the indicated populations is unknown. In the U.S. general population, the estimated background risk of major birth defect and miscarriage in clinically recognized pregnancies is 2 – 4% and 15 – 20%, respectively. 8.2 Lactation Risk Summary There is no information regarding the presence of emicizumab-kxwh in human milk, the effects on the breastfed child, or the effects on milk production. Human IgG is known to be present in human milk. 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.

Metabolism

Emicizumab-kxwh exhibited dose-proportional pharmacokinetics over a dose range of 0.3 mg/kg (0.1 times approved recommended starting dosage) to 6 mg/kg following subcutaneous administration. Following multiple subcutaneous administrations of a loading dose of 3 mg/kg emicizumab-kxwh once weekly for the first 4 weeks in hemophilia A patients, mean (± SD) trough plasma concentrations of 52.6 ± 13.6 μg/mL was achieved at Week 5.

• Nov 16, 2017BLABLA761083Genentech Inc

1. 1Haemorrhage4,29537%
2. 2No Adverse Event7756.6%
3. 3Haemarthrosis7746.6%
4. 4Off Label Use7626.5%
5. 5Fall6245.3%
6. 6Arthralgia4894.2%
7. 7Contusion3883.3%
8. 8Epistaxis3793.2%
9. 9Head Injury3382.9%
10. 10Pain3142.7%
11. 11Muscle Haemorrhage2942.5%
12. 12Weight Increased2231.9%
13. 13Joint Injury2221.9%
14. 14Limb Injury2181.9%
15. 15Injury2111.8%

The 10 most recently updated of 55 ClinicalTrials.gov registrations naming Emicizumab as an intervention. Registration is not evidence of efficacy or safety — reference crosswalk only.

• A Clinical Study to Evaluate the Effects of NXT007 Compared to Emicizumab Prophylaxis in People With Hemophilia A
  Recruiting · Phase 3 · Interventional · 360 enrolled · Hoffmann-La Roche
  NCT07416604updated 2026-06-08
• A Prospective Assessment of Bone Health in Patients With Severe Hemophilia A on Factor VIII vs Factor Mimetic Prophylaxis (Efa Emi Bone Health Study)
  Not yet recruiting · Observational · 50 enrolled · Arkansas Children's Hospital Research Institute
  NCT07582276updated 2026-06-04
• A Study to Learn About How Changing Therapy From Emicizumab to Marstacimab Affects People With the Severe Hemophilia A.
  Recruiting · Phase 1 · Interventional · 15 enrolled · Pfizer
  NCT06703606updated 2026-06-03
• A Study to Test a Medicine (Fitusiran) for Preventing Bleeds in People With Severe Hemophilia Who Previously Received Preventive Treatment With Emicizumab
  Recruiting · Phase 4 · Interventional · 20 enrolled · Sanofi
  NCT06145373updated 2026-06-02
• UPLC-MS/MS Monitoring of Emicizumab Therapy
  Active not recruiting · Observational · 100 enrolled · University Hospital, Lille
  NCT04472169updated 2026-05-20
• A Study to Assess the Efficacy and Safety of Emicizumab in Participants With Type 3 Von Willebrand Disease
  Recruiting · Phase 3 · Interventional · 75 enrolled · Hoffmann-La Roche
  NCT06998524updated 2026-05-18
• Nuwiq for Perioperative Management Of Patients With Haemophilia A on Emicizumab Regular Prophylaxis Study
  Recruiting · Phase 4 · Interventional · 28 enrolled · Octapharma
  NCT05935358updated 2026-05-12
• SAFE Study: Safety of aPCC Following Emicizumab Prophylaxis
  Recruiting · Phase 3 · Interventional · 5 enrolled · Emory University
  NCT04563520updated 2026-04-29
• A Study to Evaluate Overall Health, Physical Activity, and Joint Outcomes in Participants With Severe or Moderate Hemophilia A Without Factor VIII Inhibitors on Emicizumab Prophylaxis
  Active not recruiting · Phase 4 · Interventional · 136 enrolled · Hoffmann-La Roche
  NCT05181618updated 2026-04-27
• A Study to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Subcutaneous Emicizumab in Participants From Birth to 12 Months of Age With Hemophilia A Without Inhibitors
  Active not recruiting · Phase 3 · Interventional · 55 enrolled · Hoffmann-La Roche
  NCT04431726updated 2026-04-13

Frequently asked questions

How does Emicizumab work?

HEMLIBRA bridges activated factor IX and factor X to restore the function of missing activated factor VIII that is needed for effective hemostasis.

What is Emicizumab used for?

According to FDA labeling, Emicizumab carries indications including: 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 (congenital factor VIII deficiency) with or without factor VIII inhibitors. HEMLIBRA is a bispecific factor IXa- and factor X-directed antibody 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 (congenital factor VIII deficiency) with or without factor VIII inhibitors.. This is a reference summary of labeled uses, not medical advice or a treatment recommendation.

What class of drug is Emicizumab?

Emicizumab is classified as Other systemic hemostatics, Increased Coagulation Factor VIII Activity.

What are the brand names for Emicizumab?

Emicizumab is marketed under brand names including Hemlibra.

What are the contraindications for Emicizumab?

Emicizumab labeling lists contraindications including: None.. Always consult the full prescribing information and a clinician.