An editorial notes that despite major therapeutic advances in the treatment of this disease, global mortality is about 15%. It adds that in the patients who die, the vicious cycle of von Willebrand factor–dependent thrombi formation is most likely not disrupted fast enough by therapies targeting ADAMTS13 and its specific autoantibodies. Thus, a more direct and rapidly acting treatment approach would be to inhibit the binding of von Willebrand factor to platelets. Caplacizumab, an anti–von Willebrand factor humanized single-variable-domain immunoglobulin (Nanobody), inhibits the interaction between ultralarge von Willebrand factor multimers and platelets.
In May 2015, the company reported that it intended to seek conditional approval from the European Medicines Agency for caplacizumab in the treatment of acquired thrombotic thrombocytopenic purpura in the first half of 2017.