Prolonging the Half-Life of Factor VIII Replacement Therapy for Hemophilia A

The half-life of BIVV001 was 3 to 4 times that of recombinant factor VIII.

The major objectives of therapy in the treatment of severe hemophilia A include reducing the frequency of bleeding, maintaining joint health, and improving quality of life. A sustained increase in factor VIII activity levels may help accomplish these goals. At present, a short half-life, limited by the von Willebrand factor (VWF) chaperone effect, results in the need for frequent administration of factor VIII replacement therapy.

Now, investigators have conducted an industry-funded, open-label, phase I/IIa study to evaluate the safety of BIVV001 — a novel fusion protein designed to uncouple factor VIII from VWF and prolong its half-life — in 16 men with severe hemophilia A lacking an inhibitor. Study participants received a single intravenou…

Reviewing Author

Brady L. Stein, MD, MHS

Disclosures

Leadership Positions in Professional SocietiesAmerican Society of Hematology (Senior Editor, ASH Self-Assessment Program; guideline development)

Brady L. Stein, MD, MHS

Disclosures

Leadership Positions in Professional SocietiesAmerican Society of Hematology (Senior Editor, ASH Self-Assessment Program; guideline development)

Prolonging the Half-Life of Factor VIII Replacement Therapy for Hemophilia A

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Reviewing Author