Concizumab (commercial name, Alhemo), a monoclonal antibody was approved by the FDA on 20 December 2024 for prevention of bleeding episodes in patients with hemophilia A with factor VIII inhibitors or hemophilia B with factor IX inhibitors. It had received approval by the European Medicines Agency (EMA) few days ago on 16 December 2024 for the same indications.
Some haemophilia patients on “clotting factor medicines” for treatment their bleeding disorder condition develop antibodies (against the clotting factor medicines). The antibodies formed inhibit the action of “clotting factor medicines” making them less effective. This condition is currently treated by inducing immune tolerance through daily injections of clotting factors. Approval of Concizumab (Alhemo) provides such patients with an alternative treatment.
Concizumab is administered daily as subcutaneous injection.
Approval of Alhemo was based on evaluation of its safety and efficacy in a multi-national, multi-centre, open-label, phase 3 clinical trial. In the trial, the annualized bleeding rates (ABR) was reduced by 86% for the Alhemo treatment group compared to the no prophylaxis group.
Bleeding disorders in haemophilia are caused due to inadequacy of clotting factors. Haemophilia A is caused due to deficiency of clotting factor VIII, while haemophilia B is due to low levels of factor IX. Lack of functional factor XI is responsible for haemophilia C. These conditions are treated by infusing commercially prepared clotting factor or a non-factor product as functional replacement of the missing factor.
Octocog alfa (Advate), which is a ‘genetically engineered using DNA technology’ version of clotting factor VIII, is commonly used for the preventive as well as on-demand treatment of haemophilia A. For haemophilia B, nonacog alfa (BeneFix), which is an engineered version of clotting factor IX is commonly used.
Hympavzi (marstacimab-hncq), a human monoclonal antibody targeting “tissue factor pathway inhibitor” was recently approved as a new drug for prevention of bleeding episodes in individuals with hemophilia A or hemophilia B.
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References:
- FDA approves drug to prevent or reduce the frequency of bleeding episodes for patients with hemophilia A with inhibitors or hemophilia B with inhibitors. Posted 20 December 2024. Available at https://www.fda.gov/drugs/news-events-human-drugs/fda-approves-drug-prevent-or-reduce-frequency-bleeding-episodes-patients-hemophilia-inhibitors-or
- EMA. Alhemo – Concizumab. Available https://www.ema.europa.eu/en/medicines/human/EPAR/alhemo and https://ec.europa.eu/health/documents/community-register/html/h1881.htm
- NHS. Treatment of Hemophilia. Available at https://www.nhs.uk/conditions/haemophilia/treatment/
- CDC. Treatment of Hemophilia. Available at https://www.cdc.gov/hemophilia/treatment/index.html
Related article
- Hympavzi (marstacimab): New Treatment for Hemophilia. Scientific European. Posted 12 October 2024. Available at https://www.scientificeuropean.co.uk/medicine/hympavzi-marstacimab-new-treatment-for-hemophilia/
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