Luspatercept: First Approval

Drugs. 2020 Jan;80(1):85-90. doi: 10.1007/s40265-019-01251-5.

Abstract

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.

Publication types

  • Review

MeSH terms

  • Activin Receptors, Type II / therapeutic use*
  • Anemia / drug therapy*
  • Anemia / etiology
  • Animals
  • Drug Approval
  • Hematinics / therapeutic use*
  • Humans
  • Immunoglobulin Fc Fragments / therapeutic use*
  • Myelodysplastic Syndromes / complications
  • Myelodysplastic Syndromes / drug therapy
  • Primary Myelofibrosis / complications
  • Primary Myelofibrosis / drug therapy
  • Recombinant Fusion Proteins / therapeutic use*
  • beta-Thalassemia / complications
  • beta-Thalassemia / drug therapy

Substances

  • Hematinics
  • Immunoglobulin Fc Fragments
  • Recombinant Fusion Proteins
  • luspatercept
  • Activin Receptors, Type II