TTP is a complex disease with a long list of symptoms that confound even the most seasoned physicians. It often requires a multidisciplinary approach involving hematologists, nephrologists and other specialists, which can pose logistical challenges. The first-line option of PEX is challenging and associated with weeks of hospitalization, and patients often cite¹ the trauma of “having [their] entire blood volume passed through a machine and fed back to [them] combined with strangers’ blood”. It must be clarified that TTP is not the same as idiopathic thrombocytopenic purpura (ITP). Although both blood disorders affect platelets, they have different causes and symptoms: ITP is an autoimmune disorder that results in low platelet counts (the Syk inhibitor fostamatinib² from Grifols is approved for this disease), while TTP is caused by abnormal clotting that can lead to organ damage and other serious complications.

But hope is on the horizon with two groundbreaking treatments: Caplacizumab and TAK-755. Reducing TTP-related deaths, recurrence and thromboembolic events, Sanofi’s caplacizumab is proving to be a game changer in the TTP space. Meanwhile, Takeda’s TAK-755 promises to revolutionize TTP treatment with its targeted approach that addresses the deficient ADAMTS13 enzyme upstream of the coagulation pathway. TAK-755 has shown promise in clinical trials; it reduces the time to platelet normalization, lowers the incidence of TTP-related complications and has demonstrated a 60% reduction in thrombocytopenia events compared to the standard of care (SoC). In addition to its encouraging efficacy though, TAK-755 also has lower associated incidences of adverse events compared with the SoC. Accordingly, Takeda is seeking³ marketing authorization for TAK-755 as the first recombinant ADAMTS13 replacement therapy for chronic TTP, and with its impressive safety and efficacy, lower administration frequency and improved cost-effectiveness, it is no wonder that hematologists and patients alike eagerly await its approval.