Luspatercept

Luspatercept (trade name Reblozyl; development code ACE-536)[2] is a drug for the treatment of anemia in beta thalassemia and myelodysplastic syndromes. It was developed by Acceleron Pharma in collaboration with Celgene.[3]

Luspatercept
Clinical data
Trade namesReblozyl
Other namesACE-536, luspatercept-aamt
AHFS/Drugs.comMonograph
License data
Pregnancy
category
  • US: N (Not classified yet) [1]
    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.[4][5]

    Clinical development

    Phase III trials evaluated the efficacy of luspatercept for the treatment of anemia in the hematological disorders beta thalassemia[6] and myelodysplastic syndromes.[7][5]

    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.[8] 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.[9][10][11][12]

    In April 2020, the Committee for Medicinal Products for Human Use (CHMP) in the EU recommended luspatercept (Reblozyl) for approval. It received a positive opinion from the CHMP for the treatment of adults with transfusion-dependent anaemia associated with myelodysplastic syndromes (diseases in which the body produces large numbers of abnormal blood cells) or beta thalassaemia (a blood disorder that reduces the production of haemoglobin).[13]

    References
    1. "Luspatercept (Reblozyl) Use During Pregnancy". Drugs.com. 16 December 2019. Retrieved 27 January 2020.
    2. "Luspatercept". AdisInsight. Retrieved 15 February 2017.
    3. "Luspatercept: Our Lead Product Candidate". Acceleron Pharma. Retrieved 22 May 2017.
    4. "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.
    5. "Luspatercept". AdisInsight. Retrieved 1 February 2017.
    6. 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
    7. 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
    8. "Luspatercept". NCI Thesaurus. National Cancer Institute.
    9. "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.
    10. "Reblozyl (luspatercept-aamt) FDA Approval History". Drugs.com. Archived from the original on 13 November 2019. Retrieved 13 November 2019.
    11. "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.
    12. "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.
    13. "Meeting highlights from the Committee for Medicinal Products for Human Use (CHMP) 28-30 April 2020". European Medicines Agency (EMA). 30 April 2020. Retrieved 6 May 2020. This article incorporates text from this source, which is in the public domain.
    • "Luspatercept". Drug Information Portal. U.S. National Library of Medicine.


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