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

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

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  • 1

    Congenital thrombotic thrombocytopenic purpura (cTTP) is an autosomal recessive disorder caused by ADAMTS13 deficiency.

  • 2

    cTTP leads to microvascular thrombosis, consumptive thrombocytopenia, and microangiopathic hemolytic anemia.

  • 3

    Recombinant ADAMTS13 (rADAMTS13) is the first FDA-approved enzyme replacement therapy for managing cTTP.

  • 4

    rADAMTS13 restores cleavage of ultra-large vWF multimers, reducing platelet aggregation and thrombotic events.

  • 5

    Clinical trials show rADAMTS13 is effective and provides an alternative to plasma infusions for cTTP treatment.

Original Source(s)

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

  • by Taylor P. Robichaux, Kathryn E. Dane, P. Christopher Parish, Justin R. Arnall, Donald C. Moore

  • January 9, 2026

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