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Semin Thromb Hemost
DOI: 10.1055/a-2735-1659
Commentary

From Fresh Frozen Plasma to First-in-human: Bringing Coagulation Factor V Deficiency into Therapeutic Trials

Authors

  • Jeremy W. Jacobs 

    1   Division of Transfusion Medicine, Department of Pathology, Microbiology, and Immunology, Vanderbilt University, Nashville, Tennessee
    2   Special Coagulation Laboratory, Vanderbilt Medical Laboratories, Nashville, Tennessee

  • Sheharyar Raza

    3   Division of Hematology, University of Toronto, Toronto, Ontario, Canada
    4   Medical Affairs and Innovation, Canadian Blood Services, Ottawa, Ontario, Canada

  • Cristina A. Figueroa Villalba

    5   Department of Laboratory Medicine and Pathology, University of Washington, Seattle, Washington
    6   Department of Laboratory Medicine and Pathology, Seattle Children's Hospital, Seattle, Washington

  • Brian D. Adkins

    7   Division of Transfusion Medicine and Hemostasis, University of Texas Southwestern Medical Center, Dallas, Texas

  • Shannon C. Walker

    1   Division of Transfusion Medicine, Department of Pathology, Microbiology, and Immunology, Vanderbilt University, Nashville, Tennessee
    8   Division of Pediatric Hematology and Oncology, Department of Pediatrics, Vanderbilt University Medical Center, Nashville, Tennessee

  • Garrett S. Booth

    1   Division of Transfusion Medicine, Department of Pathology, Microbiology, and Immunology, Vanderbilt University, Nashville, Tennessee

  • Deva Sharma

    1   Division of Transfusion Medicine, Department of Pathology, Microbiology, and Immunology, Vanderbilt University, Nashville, Tennessee
    9   Division of Hematology/Oncology, Department of Medicine, Vanderbilt University Medical Center, Nashville, Tennessee

  • Allison P. Wheeler

    10   Washington Center for Bleeding Disorders, Seattle, Washington
    11   Division of Hematology/Oncology, Department of Medicine, University of Washington School of Medicine, Seattle, Washington
    12   Division of Pediatric Hematology/Oncology, University of Washington School of Medicine, Seattle, Washington
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Abstract

Congenital factor V (FV) deficiency, affecting approximately 1 in 1 million individuals worldwide, remains among the rare bleeding disorders (RBDs) without a licensed factor-specific replacement therapy. While other RBDs have successfully transitioned from plasma-based treatment to approved factor concentrates—exemplified by factor X deficiency's progression to US Food & Drug Administration (FDA)-approved Coagadex and two FDA-approved concentrates for factor XIII deficiency—FV deficiency treatment has remained unchanged for decades, relying solely on plasma and platelet transfusions. Two promising therapeutic candidates have emerged: a human plasma-derived FV concentrate demonstrating in vitro correction of severe deficiency, and an engineered activated FV (superFVa) showing potent hemostatic activity in preclinical models. This commentary outlines a pragmatic pathway to clinical trials, leveraging proven development strategies from other RBDs, existing registry infrastructure, and regulatory incentives for rare diseases. We propose phased trials combining pharmacodynamic endpoints with clinical outcomes, enabling feasible enrollment while generating decision-grade evidence. The time has come to extend modern therapeutic development to FV deficiency.

Keywords

factor V - rare bleeding disorder - hemostasis - coagulation - clinical trials


Publication History

Received: 09 September 2025

Accepted: 30 October 2025

Accepted Manuscript online:
30 October 2025

Article published online:
10 November 2025

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