Caplacizumab: First Global Approval.

Ablynx, a Sanofi Company, has developed the anti-von Willebrand factor Nanobody® caplacizumab (Cablivi™) for the treatment of acquired thrombotic thrombocytopenic purpura (aTTP). Based on positive results in phase II and III trials in patients with aTTP, caplacizumab was recently approved in the EU for the treatment of adults experiencing an episode of aTTP, in conjunction with plasma exchange and immunosuppression. This article summarizes the milestones in the development of caplacizumab leading to this first approval.