Key clinical point: The Food and Drug Administration has approved caplacizumab (Cablivi) in combination with plasma exchange and immunosuppressive therapy for the treatment of adults with acquired thrombotic thrombocytopenic purpura (aTTP).
Major finding: The FDA’s approval of caplacizumab was based on results from the phase 3 HERCULES trial, which included 145 adults with aTTP who were randomized to receive caplacizumab or placebo, in addition to plasma exchange and immunosuppression. The study’s primary endpoint was the time to platelet count response (normalization).
Study details: There was a significant reduction in time to platelet count response in the caplacizumab arm, compared with the placebo arm – 2.69 days and 2.88 days, respectively. The platelet normalization rate ratio was 1.55 (P less than .01). The most common treatment-emergent adverse events (occurring in at least 15% of patients in the caplacizumab and placebo arms, respectively) were epistaxis (32% and 3%), headache (23% and 8%), urticaria (17% and 7%), and hypokalemia (9% and 19%).
Disclosures: Cablivi is marketed by Ablynx.
FDA news release: FDA approves first therapy for the treatment of adult patients with a rare blood clotting disorder