NICE draft guidance does not recommend use of Cablivi in thrombotic thrombocytopenic purpura.- Sanofi

NICE has announced draft guidelines rejecting Cablivi (caplacizumab), from Sanofi, for use alongside plasma exchange and immunosuppression for treating an acute episode of acquired thrombotic thrombocytopenic purpura (TTP). For this condition, standard care includes immunosuppressant medicines along with plasma exchange. Data from trials shows that adding caplacizumab cuts the time needed to return blood platelet levels down to normal, and also reduces the number of plasma exchange treatments needed, along with time spent in hospital. Data suggests that the addition of caplacizumab may also reduce the long-term complications of acquired TTP as well as the risk of death during acute episodes. Despite these results, NICE claimed that the data does not consider whether adding caplacizumab improves either survival time, or quality of life in the long term. NICE also pointed out that limitations in the clinical evidence mean that the cost-effectiveness estimates are uncertain versus standard care, and as they may be higher than normally considered cost-effective, the drug is not recommended.