Voxelotor is as an oral, once-daily therapy that is being developed for treatment of SCD. It is designed to work by helping haemoglobin hold onto more oxygen as the red blood cells travel through the body. This keeps red blood cells in their normal shape and helps stop sickling. As there is a desperate need for new treatments for SCD, voxelotor, if licensed, will offer a new treatment option for patients with SCD.
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.