CABLIVI caplacizumab

Drug Class

von Willebrand factor (vWF)-directed antibody fragment

Mechanism

Caplacizumab binds to the A1 domain of von Willebrand factor (vWF), blocking its interaction with platelet GP Ib-IX-V receptors and preventing microvascular thrombosis.

Approved Indication

Treatment of adults and pediatric patients 12 years of age and older with acquired thrombotic thrombocytopenic purpura (aTTP), in combination with plasma exchange and immunosuppressive therapy.

What This Means For Patients

Cablivi (caplacizumab) is a targeted antibody fragment approved for treating acquired thrombotic thrombocytopenic purpura (aTTP), a rare blood clotting disorder. It helps prevent platelet aggregation and microvascular clot formation. It was approved by NMPA in November 2025 and is accessible under early-use schemes in the Boao Lecheng pilot zone.

Regulatory Approvals

• FDA — approved (2019) — label
• EMA — approved (2018) — source
• NMPA — approved (2025) — source

Pivotal Clinical Evidence

• HERCULES — Scully et al., N Engl J Med 2019 (NCT02553317) — DOI

Sources

• https://www.accessdata.fda.gov/drugsatfda_docs/label/2019/761107s000lbl.pdf
• https://www.ema.europa.eu/en/medicines/human/EPAR/cablivi
• https://doi.org/10.1056/NEJMoa1806311