Caplacizumab: First Global Approval

Drugs. 2018 Oct;78(15):1639-1642. doi: 10.1007/s40265-018-0989-0.


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.

Publication types

  • Review

MeSH terms

  • Clinical Trials, Phase II as Topic
  • Clinical Trials, Phase III as Topic
  • Drug Approval
  • European Union
  • Fibrinolytic Agents / pharmacology
  • Fibrinolytic Agents / therapeutic use*
  • Humans
  • Immunosuppression Therapy
  • Purpura, Thrombotic Thrombocytopenic / drug therapy*
  • Single-Domain Antibodies / pharmacology
  • Single-Domain Antibodies / therapeutic use*


  • Fibrinolytic Agents
  • Single-Domain Antibodies
  • caplacizumab

Supplementary concepts

  • Thrombotic thrombocytopenic purpura, acquired