Concizumab

Concizumab-mtci is a monoclonal antibody antagonist of endogenous Tissue Factor Pathway Inhibitor (TFPI). Through the inhibition of TFPI, concizumab-mtci acts to enhance FXa production during the initiation phase of coagulation which leads to improved thrombin generation and clot formation with the goal of achieving hemostasis in patients with Hemophilia A or B regardless of their inhibitor status.

• Alhemo is indicated for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients 12 years of age and older with: • hemophilia A (congenital factor VIII deficiency) with or without FVIII inhibitors • hemophilia B (congenital factor IX deficiency) with or without FIX inhibitors Alhemo is a tissue factor pathway inhibitor (TFPI) antagonist indicated for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients 12 years of age and older with: • hemophilia A (congenital factor VIII deficiency) with or without FVIII inhibitors • hemophilia B (congenital factor IX deficiency) with or without FIX inhibitors ( 1 )

• Alhemo is contraindicated in patients with a history of known serious hypersensitivity to Alhemo or its components or the inactive ingredients [see Warnings and Precautions ( 5.1 )and Description ( 11 )]. Alhemo is contraindicated in patients with a history of known serious hypersensitivity to Alhemo or its components or the inactive ingredients.contraindicated

Administer Alhemo by subcutaneous injection to the abdomen or thigh with daily rotation of injection sites. ( 2.2 ) Recommended dosing regimen: • Day 1: Loading dose of 1 mg/kg • Day 2: Once daily dose of 0.2 mg/kg until individualization of maintenance dose ( 2.1 ) o 4 weeks after initiation of treatment: For dose optimization, measure concizumab‑mtci plasma concentration by Concizumab Enzyme-Linked Immunosorbent Assay (ELISA) prior to administration of next scheduled dose using an FDA-authorized test for the measurement of concizumab-mtci concentration in plasma. See full Prescribing Information for important preparation and administration instructions and dosage adjustment. ( 2.1 , 2.3 , 2.4 ) 2.1 Recommended Dosage For subcutaneous use only. Alhemo should be administered once daily. Avoid missed doses. Recommended dosing regimen: • Day 1: Loading dose of 1 mg/kg • Day 2: Once daily dose of 0.2 mg/kg until individualization of maintenance dose (see below) • 4 weeks after initiation of treatment: For dose optimization measure concizumab-mtci plasma concentration by Concizumab Enzyme-Linked Immunosorbent Assay (ELISA) prior to administration of next scheduled dose using an FDA-authorized test. Information on the FDA-authorized test for the measurement of concizumab-mtci plasma concentration is available at https://www.accessdata.fda.gov/scripts/cdrh/cfdocs/cfpmn/denovo.cfm.

• Thromboembolic Events: Monitor patients for thromboembolic events. Advise patients to report signs and symptoms, and if they occur discontinue prophylaxis. ( 5.1 ) • Hypersensitivity Reactions: In the event of a severe hypersensitivity reaction, discontinue Alhemo. ( 5.2 ) • Increased Laboratory Values of Fibrin D dimer and Prothrombin Fragment 1.2: Alhemo increases values of fibrin D dimer and prothrombin fragment 1.2. ( 5.3 ) 5.1 Thromboembolic Events Alhemo may cause thromboembolic events. Venous and arterial thromboembolic events were reported in 1.9% of patients (6/320) in Alhemo clinical trials. These cases occurred in patients with multiple risk factors for thromboembolism, including the use of high doses or prolonged treatment with factor product or bypassing agent (2 of 6 patients). Risk factors for thromboembolism may include the use of high and/or frequent doses of breakthrough bleed treatments (factor products or bypassing agents) or conditions in which tissue factor is overexpressed (e.g., atherosclerotic disease, crush injury, cancer, disseminated intravascular coagulation, thrombotic microangiopathy, or septicemia). Inform Alhemo treated patients of signs and symptoms of thromboembolic events. Monitor patients for thromboembolic events. In case of suspicion of thromboembolic events, discontinue Alhemo and initiate further investigations and management strategies. 5.2 Hypersensitivity Reactions Alhemo can cause hypersensitivity reaction, including serious cases.

The following clinically significant adverse reactions are discussed in greater detail in other sections of the labeling: • Thromboembolic Events [see Warnings and Precautions ( 5.1 )] • Hypersensitivity Reactions [see Warnings and Precautions ( 5.2 )] • Increased Laboratory Values of Fibrin D-dimer and Prothrombin Fragment 1.2 [see Warnings and Precautions ( 5.3 )] The most frequently reported adverse reactions (incidence ≥5%) were injection site reactions, headache and urticaria. ( 6.1 ) To report SUSPECTED ADVERSE REACTIONS, contact Novo Nordisk Inc. at 1-800-727-6500 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 clinical trials of another drug and may not reflect the rates observed in clinical practice. The data in the WARNINGS AND PRECAUTIONS reflect exposure to Alhemo based on pooled data from clinical trials explorer3 (phase 1b), explorer4 (phase 2), explorer5 (phase 2), explorer7 (phase 3) and explorer8 (phase 3), in which a total of 320 male patients with hemophilia A with and without inhibitors and hemophilia B with and without inhibitors received at least one dose of Alhemo as routine prophylaxis. The patients were exposed for a total of 475 exposure years.

8.1 Pregnancy Risk Summary Based on its mechanism of action, Alhemo may cause fetal harm when administered to a pregnant woman [see Clinical Pharmacology ( 12.1 )] . There are no available data on Alhemo use in pregnant women to evaluate for a drug-associated risk of major birth defects, miscarriage or other adverse maternal or fetal outcomes. Animal reproduction studies have not been conducted with Alhemo. Although there are no data on concizumab-mtci, monoclonal antibodies can be actively transported across the placenta, and concizumab-mtci may cause fetal harm. It is unknown whether Alhemo can affect reproductive capacity. Alhemo should only be used during pregnancy if the potential benefit for the mother outweighs the potential risk to the fetus. The estimated background risk of birth defects and miscarriage for the indicated population is unknown. All pregnancies have a background risk of birth defect, loss, or other adverse outcomes. In the U.S. general population, the estimated background risk of major birth defect and miscarriage in clinically recognized pregnancies is 2 to 4% and 15 to 20%, respectively. 8.2 Lactation Risk Summary There is no information regarding the presence of Alhemo in either human or animal milk, the effect on the breastfed child, or the effects on milk production.

Metabolism

Peak and trough geometric mean plasma concizumab-mtci concentrations at steady state are shown in Table 3 . Following a single Alhemo loading dose of 1 mg/kg, the steady state concentrations were reached around Day 4 and remained within a stable exposure range with daily maintenance doses.

• Dec 20, 2024BLABLA761315Novo Nordisk Inc

1. 1Haemarthrosis723%
2. 2Injection Site Pain39.7%
3. 3Anti Factor Ix Antibody Increased26.5%
4. 4Condition Aggravated26.5%
5. 5Fall26.5%
6. 6Haematoma26.5%
7. 7Haematoma Muscle26.5%
8. 8Haematuria26.5%
9. 9Muscle Haemorrhage26.5%
10. 10Off Label Use26.5%
11. 11Abdominal Pain Upper13.2%
12. 12Acute Myocardial Infarction13.2%
13. 13Anaemia13.2%
14. 14Back Pain13.2%
15. 15Cardiac Arrest13.2%

The 10 most recently updated of 11 ClinicalTrials.gov registrations naming Concizumab as an intervention. Registration is not evidence of efficacy or safety — reference crosswalk only.

• Research Study to Look at How Well the Drug Concizumab Works in Your Body if You Have Haemophilia Without Inhibitors
  Active not recruiting · Phase 3 · Interventional · 156 enrolled · Novo Nordisk A/S
  NCT04082429updated 2026-06-12
• Research Study to Look at How Well the Drug Concizumab Works in Your Body if You Have Haemophilia With Inhibitors
  Active not recruiting · Phase 3 · Interventional · 134 enrolled · Novo Nordisk A/S
  NCT04083781updated 2026-06-12
• Targeting TFPI With Concizumab to Improve Haemostasis in Glanzmann Thrombasthenia Patients: an in Vitro Study
  Completed · Interventional · 20 enrolled · University Hospital, Bordeaux
  NCT06234813updated 2026-05-26
• Compassionate Use of Concizumab if You Have Haemophilia
  Available · Expanded access · Novo Nordisk A/S
  NCT04921956updated 2026-02-13
• A Research Study on How Well Concizumab Works for You if You Have Haemophilia A or B With or Without Inhibitors
  Active not recruiting · Phase 3 · Interventional · 153 enrolled · Novo Nordisk A/S
  NCT05135559updated 2025-12-30
• Post-Marketing Surveillance (All Case Surveillance) on Treatment With Alhemo® in Patients With Haemophilia A or Haemophilia B With Inhibitors
  Enrolling by invitation · Observational · 23 enrolled · Novo Nordisk A/S
  NCT06285071updated 2025-08-29
• Post-marketing Surveillance (Special Use-results Surveillance) on Treatment With Alhemo
  Enrolling by invitation · Observational · 30 enrolled · Novo Nordisk A/S
  NCT06831734updated 2025-08-29
• In Vitro Correction of Thrombin Generation by Concizumab (Anti-TFPI) for Severe Hemophilia Patients
  Completed · Observational · 20 enrolled · Centre Hospitalier Universitaire de Saint Etienne
  NCT05617209updated 2024-11-27
• A Trial Evaluating Efficacy and Safety of Prophylactic Administration of Concizumab in Patients With Severe Haemophilia A Without Inhibitors
  Completed · Phase 2 · Interventional · 36 enrolled · Novo Nordisk A/S
  NCT03196297updated 2021-11-16
• A Trial Evaluating the Efficacy and Safety of Prophylactic Administration of Concizumab in Haemophilia A and B Patients With Inhibitors
  Completed · Phase 2 · Interventional · 26 enrolled · Novo Nordisk A/S
  NCT03196284updated 2021-10-22

Frequently asked questions

How does Concizumab work?

Concizumab-mtci is a monoclonal antibody antagonist of endogenous Tissue Factor Pathway Inhibitor (TFPI). Through the inhibition of TFPI, concizumab-mtci acts to enhance FXa production during the initiation phase of coagulation which leads to improved thrombin generation and clot formation with the goal of achieving hemostasis in patients with Hemophilia A or B regardless of their inhibitor status.

What is Concizumab used for?

According to FDA labeling, Concizumab carries indications including: Alhemo is indicated for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients 12 years of age and older with: • hemophilia A (congenital factor VIII deficiency) with or without FVIII inhibitors • hemophilia B (congenital factor IX deficiency) with or without FIX inhibitors Alhemo is a tissue factor pathway inhibitor (TFPI) antagonist indicated for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients 12 years of age and older with: • hemophilia A (congenital factor VIII deficiency) with or without FVIII inhibitors • hemophilia B (congenital factor IX deficiency) with or without FIX inhibitors ( 1 ). This is a reference summary of labeled uses, not medical advice or a treatment recommendation.

What class of drug is Concizumab?

Concizumab is classified as Other systemic hemostatics, Tissue Factor Pathway Inhibitor Antagonist, Antibody Interactions, Tissue Factor Pathway Inhibitor Antagonists, Increased Coagulation Factor X Activity, Increased Coagulation Factor X Concentration.

What are the brand names for Concizumab?

Concizumab is marketed under brand names including Alhemo.

What are the contraindications for Concizumab?

Concizumab labeling lists contraindications including: Alhemo is contraindicated in patients with a history of known serious hypersensitivity to Alhemo or its components or the inactive ingredients [see Warnings and Precautions ( 5.1 )and Description ( 11 )]. Alhemo is contraindicated in patients with a history of known serious hypersensitivity to Alhemo or its components or the inactive ingredients.. Always consult the full prescribing information and a clinician.