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.
The amino acid L-glutamine has been developed as an oral powder formulation to reduce the acute complication of sickle cell disease. It is thought L-glutamine works by reducing cell inflammation and promote the cellular uptake of oxygen. If licensed, L-glutamine oral powder may offer an additional therapy option for those with sickle cell disease who currently have few effective therapies available.