NICE has drawn up draft guidelines rejecting Sanofi’s Cablivi (caplacizumab) for use alongside plasma exchange and immunosuppression for treating an acute episode of acquired thrombotic thrombocytopenic purpura (TTP).
Standard care for patients in this setting includes plasma exchange and immunosuppressant medicines, and trial results show that adding Cablivi reduces:
* the time it takes to bring platelet levels back to normal
* the number of plasma exchange treatments needed
* time in hospital and intensive care.
Adding Cablivi could also reduce the long-term complications of acquired TTP and risk of death around the time of an acute episode.
However, NICE said the trial does not consider whether adding Sanofi’s drug improves either length or quality of life over the long term.
It concluded that the ‘limitations in the clinical evidence’ mean that the cost-effectiveness estimates for Cablivi compared with standard care ‘are very uncertain’, and as they may be higher than the range normally considered a cost-effective use of NHS resources, the drug can not be recommended for treating acute acquired TTP.
Acquired TTP is a life-threatening, autoimmune-based blood clotting disorder characterised by extensive clot formation in small blood vessels throughout the body, leading to severe thrombocytopenia (very low platelet count), microangiopathic hemolytic anemia (loss of red blood cells through destruction), ischemia and widespread organ damage.
Cablivi, which was developed by Sanofi group Ablynx, won European approval in September 2018.
