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ADZYNMA is an enzyme replacement therapy (ERT) indicated for the treatment of ADAMTS13 deficiency in children and adult patients with congenital thrombotic thrombocytopenic purpura (cTTP). ADZYNMA can be used for all age groups.*, ^{1 1. ADZYNMA (rADAMTS13) EU Summary of Product Characteristics. August 2024.}

*ADZYNMA is a purified bivariant human recombinant “A disintegrin and metalloproteinase with thrombospondin motifs 13” (rADAMTS13) expressed in Chinese Hamster Ovary (CHO) cells using recombinant DNA technology (a mixture of native rADAMTS13 Q23 and variant rADAMTS13 R23 with a controlled range of the two variants ratio), referred to as rADAMTS13. ^{1 1. ADZYNMA (rADAMTS13) EU Summary of Product Characteristics. August 2024.}

ADZYNMA is the first and only therapy approved specifically for patients with congenital thrombotic thrombocytopenic purpura (cTTP) ^{1 1. ADZYNMA (rADAMTS13) EU Summary of Product Characteristics. August 2024. 2 2. Scully M, et al. Phase 3 prospective, randomized, controlled, open-label, multicenter, crossover study of recombinant ADAMTS13 in patients with congenital thrombotic thrombocytopenic purpura. Conference presentation at the International Society on Thro}

Mode of action

Discover how ADZYNMA works by watching this short video.

rADAMTS13 is a recombinant form of the endogenous ADAMTS13. ADAMTS13 is a plasma zinc metalloprotease that regulates the activity of von Willebrand factor (VWF) by cleaving large and ultra-large VWF multimers to smaller units and thereby reducing the platelet binding properties of VWF and its propensity to form microthrombi. rADAMTS13 is expected to reduce or eliminate the spontaneous formation of VWF-platelet microthrombi that leads to platelet consumption and thrombocytopenia in patients with cTTP. ^{1 1. ADZYNMA (rADAMTS13) EU Summary of Product Characteristics. August 2024.}

* ADZYNMA contains apadamtase alfa, a human recombinant “A disintegrin and metalloproteinase with thrombospondin motifs 13” (rADAMTS13). It is produced in Chinese Hamster Ovary (CHO) cells by recombinant DNA technology. ^{1 1. ADZYNMA (rADAMTS13) EU Summary of Product Characteristics. August 2024.}