Caplacizumab (Cablivi)

Key Messages

• CADTH recommends that Cablivi should not be reimbursed by public drug plans for the treatment of adults with acquired thrombotic thrombocytopenic purpura (aTTP) in combination with plasma exchange (PE) and immunosuppressive therapy.
• Evidence from clinical trials were unable to demonstrate that Cablivi improved survival and quality of life, minimized organ damage, or prevented long-term aTTP recurrence. Although results showed that Cablivi reduced time to platelet normalization compared with placebo in patients with aTTP who received PE and immunosuppressive therapy, CADTH Canadian Drug Expert Committee was unable to determine how this correlated with the previously mentioned clinical outcomes.
• Evidence from an integrated analysis of clinical trials and several sources of real-world evidence were unable to clearly demonstrate whether Cablivi provides benefits when added to PE and immunosuppressive therapy due to limitations associated with the methodology.
• Based on the evidence reviewed, it remains uncertain whether Cablivi meets the needs identified by patients of improving survival and quality of life, preventing disease complications, reducing the need for PE, and preventing disease recurrence.