Crizanlizumab is a medicine under development for the preventative treatment of SCD painful crisis. Circulating platelets (type of cells in the blood that has a role in blood clotting and stopping of bleeding) in SCD patients express higher levels of P-selectin, a protein that drives the blockage of blood vessels. Crizanlizumab acts by blocking P-selectin and thus can prevent blockage in small blood vessels and maintain blood flow. Therefore, if licensed, crizanlizumab will offer a new treatment option for patients with sickle cell-related pain crises.
Emapalumab is a type of drug called a monoclonal antibody, under development for the treatment of primary HLH in children. It acts by neutralising IFNγ activity produced by over‐stimulated cells of the immune system. It is administered as an intravenous infusion and may be used in addition to current treatment.