Etavopivat for treating sickle cell disease in adults and adolescents

Etavopivat is being developed as a treatment for adults and adolescents with sickle cell disease (SCD). SCD is a condition that causes red blood cells (RBCs) to become abnormally shaped, which limits their ability to carry oxygen around the body. The abnormal blood cells build up causing blockages in blood vessels. This leads to low healthy RBCs (known as anaemia) and episodes of pain called pain crises. SCD symptoms can start from a few months of age. Symptoms include tiredness and shortness of breath. SCD is caused by inheriting (passed down from parents) the sickle cell gene. Current treatments focus on treating symptoms rather than the route cause. Therefore, there is a need for better treatments that prevent pain crises, reduce complications, and improve quality of life.