Caplacizumab (Cablivi) is a selective bivalent anti -von Willebrand factor nanobody indicated for the treatment of adults with acquired thrombotic thrombocytopenic purpura (aTTP) in combination with plasma exchange (PEX) and immunosuppressive therapy. Caplacizumab is available in single or multi-pack kits (seven administrations) containing an 11 mg vial of caplacizumab powder, a pre-filled syringe of sterile water for injection, a vial adaptor, a needle, and two alcohol swabs. The recommended loading dose for caplacizumab is 11 mg by IV injection at least 15 minutes prior to PEX, followed by an 11 mg subcutaneous injection after completion of PEX on that day. Subsequently, 11 mg of caplacizumab should be administered as a daily maintenance dose by subcutaneous injection following PEX administration for the duration of daily PEX therapy, then once daily for 30 days following the last daily PEX treatment. If, after the initial treatment course, signs of persistent underlying disease such as suppressed a disintegrin and metalloproteinase with a thrombospondin type 1 motif, member 13 (ADAMTS13) activity levels remain present, treatment may be extended for a maximum of 28 days. No data on re-treatment with caplacizumab are available.

Contents

• Abbreviations
• Executive Summary
  • Background
  • Summary of Identified Limitations and Key Results
  • Conclusions
• Information on the Pharmacoeconomic Submission
  • Summary of the Sponsor’s Pharmacoeconomic Submission
  • Sponsor’s Base Case
  • Summary of Sponsor’s Sensitivity Analyses
  • Limitations of Sponsor’s Submission
  • CADTH CDR Reanalyses
  • Issues for Consideration
  • Patient Input
  • Conclusions
• Appendix 1. Cost Comparison
• Appendix 2. Summary of Key Outcomes
• Appendix 3. Additional Information
• Appendix 4. Summary of Other HTA Reviews of Drug
• Appendix 5. Reviewer Worksheets
• References

Version: Final (with redactions)

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