Recombinant ADAMTS13: An Enzyme Replacement Therapy for the Management of Congenital Thrombotic Thrombocytopenic Purpura

Congenital thrombotic thrombocytopenic purpura (cTTP) is a rare autosomal recessive condition that causes deficiency of the von Willebrand factor (vWF)-cleaving metalloprotease, ADAMTS13. Recombinant ADAMTS13 (rADAMTS13) is the first FDA-approved product for management of cTTP. This article reviews the pharmacology, pharmacokinetics, efficacy, safety, dosing, administration, and implications for advanced practitioners of rADAMTS13.

• By

• Taylor P. Robichaux

• Kathryn E. Dane

• P. Christopher Parish

• Justin R. Arnall

• Donald C. Moore

• January 9, 2026

• 15 min

• Jadpro