Add like
Add dislike
Add to saved papers

Concizumab: a novel anti-TFPI therapeutic for hemophilia.

Blood Advances 2021 January 13
Concizumab is a novel subcutaneous prophylactic therapy for hemophilia. It is a hemostatic rebalancing agent that binds to the Kunitz-2 domain of tissue factor pathway inhibitor (TFPI), one of the molecules that contributes to downregulation of coagulation thereby preventing TFPI from binding to and blocking the factor Xa (FXa) active site. When the TFPI inhibitory activity is decreased, sufficient FXa is produced by the FVIIa-tissue factor complex to achieve hemostasis. On the basis of this mechanism of action, concizumab is expected to be equally effective in hemophilia A and B, regardless of inhibitor status. Moreover, the concizumab mechanism of action does not interfere with the regulation of coagulation downstream of TFPI. Results from 2 phase 2 trials in patients with hemophilia A or B with and without inhibitors demonstrated that concizumab had a favorable safety profile, with no deaths, no thromboembolic events, and no adverse events leading to withdrawal. Clinical proof of concept in prevention of bleeding episodes was confirmed in both concizumab phase 2 trials across all hemophilia subtypes assessed, with a statistically significant and clinically relevant reduction in annualized bleeding rates observed in inhibitor patients compared to those who received on-demand treatment. On the basis of phase 2 results, the US Food and Drug Administration granted concizumab Breakthrough Therapy designation for hemophilia B with inhibitor patients, a rare and vulnerable patient subgroup that currently has the highest unmet medical need. In the ongoing concizumab phase 3 trials, an optimized dosing regimen will be administered in patients with hemophilia A or B with and without inhibitors.

Full text links

We have located links that may give you full text access.
Can't access the paper?
Try logging in through your university/institutional subscription. For a smoother one-click institutional access experience, please use our mobile app.

For the best experience, use the Read mobile app

Mobile app image

Get seemless 1-tap access through your institution/university

For the best experience, use the Read mobile app

All material on this website is protected by copyright, Copyright © 1994-2024 by WebMD LLC.
This website also contains material copyrighted by 3rd parties.

By using this service, you agree to our terms of use and privacy policy.

Your Privacy Choices Toggle icon

You can now claim free CME credits for this literature searchClaim now

Get seemless 1-tap access through your institution/university

For the best experience, use the Read mobile app