Luspatercept: First Approval.

Luspatercept (REBLOZYL®) is an erythroid maturation agent developed by Acceleron Pharma and Celgene Corporation for the treatment of anaemia associated with myelodysplastic syndromes, myelofibrosis and beta-thalassaemia. Based primarily on the results of the phase III BELIEVE trial, subcutaneous luspatercept was recently approved in the USA for the treatment of anaemia associated with beta-thalassaemia. This article summarizes the milestones in the development of luspatercept leading to this first approval.