New Technologies in Treatment 2024

This year’s event took place in Helsinki, Finland, and three representatives attended on behalf of the Czech Republic. Each EHC member organization can nominate one representative from a patient organization and one medical professional. The Czech Republic was represented by Martin Žídek, Vice-President of the Czech Haemophilia Society (CHS), and MUDr. Eva Drbohlavová from the Liberec Haemophilia Center, who accepted the medical nomination. Additionally, Associate Professor MUDr. Jan Blatný, Ph.D., participated as a speaker and panelist, serving as a member of the EHC Medical Advisory Group and the current president of the European Association for Haemophilia and Allied Disorders (EAHAD).

During the event, leading physicians and researchers presented the latest research findings, evaluations of currently available medications, and updates on drugs in clinical trials or pre-clinical stages. Below is an overview of the key topics discussed this year.

Haemophilia A

Super Long-Acting Drug

For the treatment of congenital F VIII deficiency, much attention is focused on a new drug with an extremely prolonged effect entering clinical practice in Europe this year. The active ingredient, efanesoctocog alfa, aims to maintain F VIII levels above 40% for approximately four days and not drop below 10-15% on the seventh day after administration. This weekly intravenous drug promises an unprecedented combination of bleeding protection and convenience. Early feedback indicates no significant adverse effects. Moreover, the XTEND-ed study on prophylaxis in children showed no cases of inhibitor development against F VIII.

Non-Factor Therapy

Emicizumab, a non-factor therapy, mimics F VIII activity without directly replacing it. Administered subcutaneously every one to four weeks, it provides stable levels equivalent to 10-15% of F VIII. It is effective for patients with or without inhibitors and has transformed severe haemophilia A into a milder form. Studies like HAVEN 7 confirm its efficacy and safety even in infants. Recent data from 2012-2023 involving 24,000 patients also highlight its safety with minimal thrombotic risk.

Trials are exploring emicizumab’s use in moderate or mild haemophilia A and von Willebrand disease (VWD) type 3, with promising results. For acquired haemophilia A, early emicizumab use shows potential for managing bleeding and improving prognosis. Going forward, extended half-life factor therapies or emicizumab will likely become first-line prophylaxis, tailored to individual patient needs. Experimental therapies like Mim8 and NXT007 aim for enhanced efficacy with lower doses, expected by 2027 and 2030, respectively.

Rebalancing Therapy

Rebalancing agents, while effective, may see limited use in haemophilia A due to the availability of superior alternatives. Details on these therapies can be found under the haemophilia B section.

Gene Therapy

Gene therapy research for haemophilia A continues but has yet to match the promising results seen in haemophilia B.

Emerging Technologies

A groundbreaking development is Inno8, a non-factor therapy tablet taken daily. Though still in early research stages, it holds immense future potential.

Haemophilia B

Rebalancing Therapy

Rebalancing agents for haemophilia B, such as concizumab and marstacimab, offer subcutaneous administration options. While results are promising, their use may be limited to specific cases, such as patients with inhibitors. Factor therapies with extended half-life remain the primary choice due to their long dosing intervals and efficacy.

Extended Half-Life Drugs

Pegylated extended half-life drugs are shown to stabilize polymer levels in the body over time, alleviating concerns about long-term accumulation and confirming their safety.

Gene Therapy

Gene therapy shows exceptional promise for haemophilia B, with several treatments recently approved in Europe. Early data suggest stable F IX expression levels even seven years post-treatment.

Von Willebrand Disease

Monoclonal Antibodies

Research focuses on monoclonal antibodies like VGA039, which show safety and efficacy in early trials, with potential weekly subcutaneous administration. Emicizumab is also being applied to VWD type 3, demonstrating significant reductions in bleeding and improved quality of life for patients in the Czech Republic.

Conclusion

This year’s workshop showcased open discussions among global experts, offering hope for continued advances in treating haemophilia and VWD. Events like this foster collaboration between patient advocates and medical professionals. As treatment options expand, patient organizations play a crucial role in disseminating information, ensuring shared decision-making between doctors and patients.

We extend our gratitude to the European Haemophilia Consortium for covering all travel expenses for the Czech representatives.