Luspatercept

Luspatercept, sold under the brand name Reblozyl, is a medication used for the treatment of anemia in beta thalassemia and myelodysplastic syndromes. It was developed by Acceleron Pharma in collaboration with Celgene.[1]

Luspatercept
Clinical data
Trade namesReblozyl
Other namesACE-536, luspatercept-aamt
AHFS/Drugs.comMonograph
MedlinePlusa620043
License data
Routes of
administration		Subcutaneous injection
ATC code
Legal status
Legal status
Identifiers
CAS Number
PubChem CID
DrugBank
ChemSpider
  • none
UNII
KEGG
Chemical and physical data
FormulaC3350H5070N906O1044S38
Molar mass75958.99 g·mol−1

The U.S. Food and Drug Administration (FDA) awarded orphan drug status in 2013, and fast track designation in 2015, for both indications.[2] The U.S. Food and Drug Administration (FDA) considers it to be a first-in-class medication.[3]

Clinical development

Phase III trials evaluated the efficacy of luspatercept for the treatment of anemia in the hematological disorders beta thalassemia[4] and myelodysplastic syndromes.[5][6]

Structure and Function

Luspatercept is a recombinant fusion protein derived from human activin receptor type IIb (ActRIIb) linked to a protein derived from immunoglobulin G.[7] It binds TGF (transforming growth factor beta) superfamily ligands to reduce SMAD signaling.

History

The U.S. Food and Drug Administration (FDA) granted approval for luspatercept–aamt in November 2019, for the treatment of anemia (lack of red blood cells) in adult patients with beta thalassemia who require regular red blood cell (RBC) transfusions.[8][9][10][11]

Luspatercept was approved for medical use in the European Union in June 2020.[12]

References
  1. "Luspatercept: Our Lead Product Candidate". Acceleron Pharma. Retrieved 22 May 2017.
  2. "FDA Fast Track Designation Granted to Luspatercept for the Treatment of Patients with Beta-Thalassemia" (Press release). Acceleron/Celgene. 18 May 2015. Retrieved 22 May 2017 – via Business Wire.
  3. "New Drug Therapy Approvals 2019". U.S. Food and Drug Administration. 31 December 2019. Retrieved 15 September 2020.
  4. Clinical trial number NCT02604433 for "An Efficacy and Safety Study of Luspatercept (ACE-536) Versus Placebo in Adults Who Require Regular Red Blood Cell Transfusions Due to Beta (β) Thalassemia (BELIEVE)" at ClinicalTrials.gov
  5. Clinical trial number NCT02631070 for "A Study of Luspatercept (ACE-536) to Treat Anemia Due to Very Low, Low, or Intermediate Risk Myelodysplastic Syndromes (MEDALIST)" at ClinicalTrials.gov
  6. "Luspatercept". AdisInsight. Retrieved 1 February 2017.
  7. "Luspatercept". NCI Thesaurus. National Cancer Institute.
  8. "FDA approves first therapy to treat patients with rare blood disorder". U.S. Food and Drug Administration (FDA) (Press release). 8 November 2019. Archived from the original on 13 November 2019. Retrieved 13 November 2019. This article incorporates text from this source, which is in the public domain.
  9. "Reblozyl (luspatercept-aamt) FDA Approval History". Drugs.com. Archived from the original on 13 November 2019. Retrieved 13 November 2019.
  10. "FDA Approves Reblozyl (luspatercept-aamt) for the Treatment of Anemia in Adults With Beta Thalassemia Who Require Regular Red Blood Cell Transfusions". Celgene (Press release). 8 November 2019. Archived from the original on 13 November 2019. Retrieved 13 November 2019.
  11. "Drug Trials Snapshots: Reblozyl". U.S. Food and Drug Administration (FDA). 8 November 2019. Retrieved 26 January 2020. This article incorporates text from this source, which is in the public domain.
  12. "Reblozyl EPAR". European Medicines Agency (EMA). 28 April 2020. Retrieved 26 September 2020.
  • "Luspatercept". Drug Information Portal. U.S. National Library of Medicine.


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