Crizanlizumab for the prevention of recurrent vaso-occlusive crises in children with sickle cell disease

Crizanlizumab has been developed for the prevention of recurrent vaso-occlusive crises in sickle cell disease (SCD). SCD is an inherited condition that causes red blood cells to become rigid and misshapen, often in a sickle shape. As these cells cannot move freely, this can cause blockages known as vaso-occlusion which leads to insufficient oxygen being delivered to tissues and organs, causing ischaemic injuries and excruciating pain. The frequency, severity and duration of these crises varies. There are very few NICE recommended treatment options for SCD and there are none for the paediatric population. This, therefore, is an area of significant unmet need.