Shots:

• The EC has approved Adzynma (recombinant ADAMTS13) to treat ADAMTS13 deficiency in cTTP adults & children. Adzynma is also being assessed under P-IIb study for immune-mediated thrombotic thrombocytopenic purpura (iTTP)
• Approval was based on the P-III study assessing Adzynma to treat cTTP, published in the NEJM, with patients receiving Adzynma (40IU/kg, IV, weekly) or plasma-based therapy for mos.1-6 (period 1), switched treatments for mos.7-12 (period 2) & then Adzynma for mos.13-18 (period 3)
• Study depicted 0 (Adzynma) vs 1 (plasma-based therapy) acute TTP event and 1 vs 7 (in 6 patients) subacute TTP events in periods 1 & 2. Efficacy data from period 3 was consistent with periods 1 & 2