Efficacy

Efficacy of Cablivi (caplacizumab) in Acquired Thrombotic Thrombocytopenic Purpura (aTTP)

Cablivi (caplacizumab) is a nanobody that targets von Willebrand factor (vWF), a protein involved in blood clotting. It has been approved for use in the treatment of adults with acquired thrombotic thrombocytopenic purpura (aTTP), a rare blood disorder characterized by blood clot formation in small blood vessels throughout the body, leading to thrombocytopenia, microangiopathic hemolytic anemia, and organ damage. Clinical trials have demonstrated that Cablivi, when used in conjunction with plasma exchange and immunosuppression, significantly reduces the time to platelet count normalization, which is a crucial marker of treatment response in aTTP. This reduction in time to platelet count recovery is associated with a decrease in the occurrence of aTTP-related deaths, aTTP recurrence, and major thromboembolic events.

The HERCULES study, a phase III clinical trial, showed that patients treated with Cablivi experienced a 74% reduction in the composite endpoint of aTTP-related death, recurrence of aTTP, or at least one major thromboembolic event, compared to those receiving placebo. The study also found that more patients treated with Cablivi achieved remission and that Cablivi treatment resulted in a lower rate of aTTP recurrence during the study period. These findings underscore the efficacy of Cablivi in improving patient outcomes in aTTP.

Efficacy of Adzynma (recombinant ADAMTS13) in Acquired Thrombotic Thrombocytopenic Purpura (aTTP)

Adzynma is the trade name for recombinant ADAMTS13, an enzyme that is deficient in patients with congenital TTP and can be reduced in acquired TTP due to autoantibodies. While recombinant ADAMTS13 is not yet commercially available or approved for the treatment of aTTP, it has shown promise in early clinical studies. In acquired TTP, the presence of autoantibodies inhibits the activity of ADAMTS13, leading to the accumulation of ultra-large von Willebrand factor multimers and subsequent platelet aggregation and thrombosis. By supplementing with recombinant ADAMTS13, it is hypothesized that the pathological process can be counteracted, potentially reducing the severity and duration of the disease.

Current research on recombinant ADAMTS13 is focused on its potential to become a targeted therapy for aTTP. Although data from large-scale clinical trials are not yet available, early phase studies suggest that recombinant ADAMTS13 could be beneficial in restoring enzyme activity, reducing the need for plasma exchange, and improving patient outcomes. As research progresses, recombinant ADAMTS13 may offer a new therapeutic option for the management of aTTP, particularly for patients who are refractory to or have contraindications for plasma exchange therapy.