Cablivi

Cablivi is the brand name for caplacizumab, a nanobody-based therapeutic developed by Ablynx (a Sanofi company) for the treatment of acquired thrombotic thrombocytopenic purpura (aTTP). It is used in adults in conjunction with plasma exchange and immunosuppressive therapy to reduce the time to platelet count normalization and to prevent ongoing microvascular thrombosis, TTP-related death, and relapse.

Mechanism of action

Caplacizumab targets the A1 domain of von Willebrand factor (vWF), blocking the interaction between ultra-large vWF multimers and platelets. By inhibiting this adhesion, the drug reduces formation of microthrombi in small vessels, helping to control the acute phase of aTTP when platelet consumption is high.

Medical use and regulatory status

Cablivi is indicated for adults with acquired TTP in addition to plasma exchange and immunosuppressive therapy. It has been approved in the European Union (2018) and the United States (2019), with labeling that notes the need for continuation alongside standard therapy for a defined period and consideration of discontinuation based on clinical response and regulatory guidance.

Administration and regimen

Caplacizumab is administered as a subcutaneous injection. It is started in parallel with plasma exchange and continued as part of the overall treatment plan, with dosing and duration determined by the treating clinician according to current regulatory labeling and patient response.

Safety and adverse effects

Bleeding is the most common adverse event associated with caplacizumab, ranging from minor mucocutaneous bleeding to more significant hemorrhage. Other reported effects include headache and fever. Because the drug interferes with hemostasis, clinicians monitor bleeding risk and manage procedures accordingly; the drug may be discontinued if major bleeding occurs or per clinical judgment.

See also: thrombotic thrombocytopenic purpura, von Willebrand factor, nanobody therapy.