Luspatercept seeks marketing authorization for the treatment of β-thalassaemia

A marketing authorization application (MAA) has been submitted to the European Medicines Agency (EMA) for luspatercept for the treatment of anaemia related to myelodysplastic syndromes and β-thalassaemia. Luspatercept (Celgene and  Acceleron Pharma Inc.) is a first-in-class erythroid maturation agent that is designed to regulate late-stage red blood cell maturation. The MAA for the agent is for adult patients with β-thalassemia-associated anaemia who require blood transfusions. In the phase 3 BELIEVE clinical trial, adult β-thalassaemia patients randomized to the luspatercept arm had a statistically and clinically meaningful reduction in transfusion burden during any 12 or 24 weeks in the study period with minor side effects, according to data presented at ASH 2018. A Phase 2 trial in non-transfusion-dependent β-thalassemia (BEYOND) is ongoing. See Celgene's Press Release here.