concizumab

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Ligands
concizumab

GtoPdb Ligand ID: 11182

Synonyms: Alhemo® | Anti-TFPI | concizumab-mtci | mab2021 | NN7415

concizumab is an approved drug (Health Canada (2023), FDA (2024))

Comment: Concizumab is a clinical stage anti-tissue factor pathway inhibitor (TFPI) monoclonal antibody. It is a humanised IgG4 construct that was developed by Novo Nordisk as a novel therapeutic for genetic bleeding disorders [2]. Concizumab binds to the Kunitz-type protease inhibitor (KPI) 2 domain of TFPI and blocks its interaction with the active site of FXa [1]. TFPI is an endogenous anticoagulant protein whose pharmacological inhibition restores hemostasis [7].

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Summary of Clinical Use
Concizumab was advanded to clinical evaluation as a prophylactic treatment for both hemophilia A and B. Non-fatal thrombotic events in Phase 3 study participants led to the suspension of concizumab trials in March 2020. By August 2020 the clinical hold on the trials was lifted and the trials were allowed to resume with new safety measures and guidelines in place. The EMA's orphan drug designation for the treatment of hemophilia B (issued in 2017) was withdrawn in December 2022. Concizumab was granted its first global approval by Health Canada in March 2023; for the treatment of hemophilia B [5]. EMA and FDA approvals for this indication followed in late 2024.

Summary of Clinical Use

Concizumab was advanded to clinical evaluation as a prophylactic treatment for both hemophilia A and B. Non-fatal thrombotic events in Phase 3 study participants led to the suspension of concizumab trials in March 2020. By August 2020 the clinical hold on the trials was lifted and the trials were allowed to resume with new safety measures and guidelines in place. The EMA's orphan drug designation for the treatment of hemophilia B (issued in 2017) was withdrawn in December 2022. Concizumab was granted its first global approval by Health Canada in March 2023; for the treatment of hemophilia B [5]. EMA and FDA approvals for this indication followed in late 2024.

Clinical Trials
Clinical Trial ID	Title	Type	Source	Comment	References
NCT04082429	Research Study to Look at How Well the Drug Concizumab Works in Your Body if You Have Haemophilia Without Inhibitors	Phase 3 Interventional	Novo Nordisk A/S	Known as the EXPLORER 6 study.
NCT04083781	Research Study to Look at How Well the Drug Concizumab Works in Your Body if You Have Haemophilia With Inhibitors	Phase 3 Interventional	Novo Nordisk A/S	Known as the EXPLORER 7 study.
NCT03196284	A Trial Evaluating the Efficacy and Safety of Prophylactic Administration of Concizumab in Haemophilia A and B Patients With Inhibitors	Phase 2 Interventional	Novo Nordisk A/S		6
NCT03196297	A Trial Evaluating Efficacy and Safety of Prophylactic Administration of Concizumab in Patients With Severe Haemophilia A Without Inhibitors	Phase 2 Interventional	Novo Nordisk A/S		6