Caplacizumab

Caplacizumab (INN; trade name Cablivi) is a bivalent single-domain antibody (VHH) designed for the treatment of thrombotic thrombocytopenic purpura (TTP) and thrombosis.

This drug was developed by Ablynx NV. On 30 August 2018, it was approved in the European Union for the "treatment of adults experiencing an episode of acquired thrombotic thrombocytopenic purpura (aTTP), in conjunction with plasma exchange and immunosuppression".

It is an anti-von Willebrand factor humanized immunoglobulin. It acts by blocking platelet aggregation to reduce organ injury due to ischemia. Results of the phase II TITAN trial have been reported.

Common adverse effects include injection site reactions, reported in 3–6% of patients in the HERCULES and TITAN trials.

In February 2019, caplacizumab-yhdp (Cablivi, Ablynx NV) was approved in the United States for the treatment of adults with acquired thrombotic thrombocytopenic purpura (aTTP). The drug is used in combination with plasma exchange and immunosuppressive therapy. The U.S. Food and Drug Administration (FDA) considers it to be a first-in-class medication.

While it has been adopted for upfront use in TTP treatment at a number of institutions, its cost-effectiveness has been questioned. Use of caplacizumab without plasmapheresis has been reported in select patients. The MAYARI study was designed to evaluate the effectiveness of this option.