Plasma-derived human factor X concentrate for on-demand and perioperative treatment in factor X-deficient patients: pharmacology, pharmacokinetics, efficacy, and safety

ABSTRACT

Introduction: Hereditary factor X (FX) deficiency is a rare autosomal recessive bleeding disorder characterized mainly by mild-to-severe bleeding into the mucous membranes, muscles or joints. Previously, treatment options for hereditary FX deficiency were limited mostly to products that may not specify FX content (i.e. fresh frozen plasma and prothrombin complex concentrates) and that have associated safety concerns. To meet the need for a single-factor replacement therapy specifically for use in FX-deficient patients, a high-purity, high-potency, human plasma-derived FX concentrate (pdFX; Coagadex®; Bio Products Laboratory, Elstree, UK) has been developed and approved for treatment of perioperative bleeding and on-demand treatment in FX-deficient patients.

Areas covered: The pharmacology, efficacy, and safety of pdFX are discussed, with a review of preclinical studies and clinical trial data that led to regulatory approval of pdFX in the United States and Europe.

Expert opinion: As the first single-factor replacement therapy indicated for hereditary FX deficiency, pdFX is a safe and efficacious treatment option in patients aged ≥12 years with hereditary FX deficiency. Clinical studies of pdFX provide a dosing regimen for use in cases of bleeding episodes, surgery, and prophylaxis. Further studies are ongoing regarding use of pdFX long term and in patients aged ≤12 years.

KEYWORDS:

• Clotting factor concentrate
• efficacy
• factor X deficiency
• orphan drug
• pharmacokinetics
• rare bleeding disorders
• safety

Acknowledgments

The author would like to thank Bio Products Laboratory Ltd (Elstree, UK) for its review of the pdFX data included in this manuscript.

Declaration of interest

A Shapiro is an advisory board member for Baxter BioScience, Biogen, Novo Nordisk; consultant for Baxter BioScience, Biogen, Kedrion Biopharma, Novo Nordisk, ProMetic Life Sciences; speaker’s bureau participant for Biogen; member of the International Network for Pediatric Hemophilia funded by Bayer Healthcare; member of the Novo Nordisk Hemophilia Foundation funded by Novo Nordisk; contributed to clinical research protocols for Baxter BioScience, Bayer Healthcare, Biogen, CSL-Behring, Daiichi Sankyo, Kedrion Biopharma, Novo Nordisk, Octapharma, OPKO, ProMetic Life Sciences, PTC Therapeutics, Selexys; all grant/research funding and consultant fees associated with the above activities are paid to the Indiana Hemophilia & Thrombosis Center. The author has 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.

Writing assistance was utilized in the preparation of this manuscript; it was funded by Bio Products Laboratory Ltd (Elstree, UK) and carried out by Hannah Mace, MSc, and Morgan C. Hill, PhD (QXV Communications, Haddam, CT, USA), who drafted and revised the manuscript based on input from authors, and Paula Stuckart (QXV Communications) who copyedited and styled the manuscript per journal requirements.

Additional information

Funding

Bio Products Laboratory Ltd (Elstree, UK) provided funding for medical writing and editorial support in the development of this manuscript.