Emerging drugs for the treatment of hemophilia A and B

ABSTRACT

Introduction: Replacement therapy with clotting factor concentrates is the most appropriate and effective way to treat bleedings of Hemophilia A&B to prevent chronic arthropathy. Unfortunately, the short half-life (HL) of FVIII/IX concentrates obliges the patients to receive frequent infusions, a big concern for children. The development of inhibitors in about 30–45% of hemophilia A and in 3–5% of hemophilia B patient is the major adverse event of replacement therapy.

Areas covered: In the last few years, new rFIX have been developed with HL. New rFVIII concentrates are displaying small increase of PK characteristics. The new bio-engineering methods allowed the production of molecules fused with Fc fragment of IgG or Albumin or linked to PEG. A new approach to improve hemostasis is represented by Mab against TFPI and small RNA interfering with Antithrombin synthesis. Another innovative drug seems to be the new bi-specific antibody which mimics FVIII function in linking FXa and FX to tenase production.

Expert opinion: The emerging drugs for hemophilia treatment seem to be very promising. The extended half-life will improve the adherence of patients to therapy. Accurate post-marketing surveillance studies will be necessary to check the efficacy, safety and immunogenicity of these new molecules.

KEYWORDS:

• ACE910
• ANL-AT3
• Concizumab
• N8-G
• N9-GP
• pharmacokinetics
• rFIXFc
• rFVIIIFc
• rIX-FP
• rVIII-SingleChain

Declaration of interest

M Morfini has acted as a paid consultant to Baxter, Novo Nordisk, Pfizer and has received speaker fees from CSL Behring, Kedrion, Novo Nordisk and Octapharma Symposia. E Zanon has received speaker fees/acted as a consultant for Bayer, Baxalta, CSL Behring, Grifols, Pfizer and Novo Nordisk. The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed.

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Funding

This paper has not been funded