Apadamtase alfa

Apadamtase alfa is a recombinant human form of the enzyme ADAMTS13 (a disintegrin and metalloproteinase with thrombospondin motifs 13), developed as an enzyme replacement therapy for patients with congenital thrombotic thrombocytopenic purpura (cTTP). It works by replacing deficient or absent ADAMTS13, a plasma zinc metalloprotease that regulates the activity of von Willebrand factor (VWF) by cleaving large VWF multimers into smaller units. This action helps prevent abnormal blood clot formation characteristic of cTTP. Apadamtase alfa is administered intravenously and is indicated for both prophylactic and on-demand treatment in adults and children with cTTP. The drug was developed by Takeda under license from KM Biologics, approved in the US in November 2023, and in the EU in August 2024[1][2][6][7].

Brand names

Adzynma

Other names

Recombinant ADAMTS13Recombinant-A disintegrin and metalloproteinase with thrombospondin type 1 motifs 13