Factor IX stimulants in preclinical and early phase trials for hemophilia B treatment

ABSTRACT

Introduction

Hemophilia B is a X-linked rare inherited bleeding disorder characterized by coagulation factor IX (FIX) deficiency. Therapy for hemophilia B is aimed at replacing the FIX deficiency by means of several plasma-derived or recombinant FIX products. The recent availability of recombinant FIX concentrates with a prolonged FIX half-life represented a great technological advance, permitting more spaced drug infusions and reducing treatment burden among hemophilia B patients.

Areas covered

This review summarizes the main preclinical and phase 1/2 studies investigating the innovative hemostatic products for hemophilia B replacement therapy.

Expert opinion

The significant recent technological advantages in the treatment of hemophilia B has led to the development of innovative FIX products aimed at further extending FIX half-life and using increasingly effective and convenient modes of administration. These novel hemostatic agents, currently at preclinical or early clinical phase of development, carry the potential of improving patients’ health status and quality of life. Continuous research is anyway needed to offer to such patients a concrete chance of conducting a normal existence, like to non-affected age-matched individuals.

KEYWORDS:

• hemophilia B
• factor IX
• bleeding
• novel hemostatic products
• therapy

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Article highlights

• Hemophilia B is a rare congenital bleeding disorder affecting both children and adults with a significant negative social and clinical impact.

• Prophylaxis is currently the mainstay of treatment of severe hemophilia B patients, reducing bleeding and improving joint health, quality of life, and survival.

• To improve patients’ adherence to treatment and quality of life, several treatments have been implemented in the last years, including extended half-life recombinant FIX products, non-replacement and gene therapies.

• Among investigational recombinant FIX products, the extended half-life dalcinonacog alfa is the most promising, carrying the advantage of subcutaneous administration.

Declaration of interest

The authors have no 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. This includes employment, consultancies, honoraria, stock ownership or options, expert testimony, grants or patents received or pending, or royalties.

Reviewer disclosures

Peer reviewers on this manuscript have no relevant financial or other relationships to disclose.

Additional information

Funding

This paper was not funded.