Luspatercept


Luspatercept is a drug for the treatment of anemia in beta thalassemia and myelodysplastic syndromes. It was developed by Acceleron Pharma in collaboration with Celgene.
The U.S. Food and Drug Administration awarded orphan drug status in 2013 and fast track designation in 2015 for both indications.

Clinical development

Phase III trials evaluated the efficacy of luspatercept for the treatment of anemia in the hematological disorders beta thalassemia and myelodysplastic syndromes.

Structure and Function

Luspatercept is a recombinant fusion protein derived from human activin receptor type IIb linked to a protein derived from immunoglobulin G. It binds TGF superfamily ligands to reduce SMAD signaling.

History

The U.S. Food and Drug Administration granted approval for luspatercept–aamt in November 2019, for the treatment of anemia in adult patients with beta thalassemia who require regular red blood cell transfusions.
In April 2020, the Committee for Medicinal Products for Human Use in the EU recommended luspatercept for approval. It received a positive opinion from the CHMP for the treatment of adults with transfusion-dependent anaemia associated with myelodysplastic syndromes or beta thalassaemia.