Luspatercept

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Luspatercept
Clinical data
Routes of
administration
Subcutaneous injection
Identifiers
CAS Number
ChemSpider
  • none
UNII
Chemical and physical data
FormulaC3350H5070N906O1044S38
Molar mass75958.99 g·mol−1

Luspatercept (ACE-536)[1] is an experimental drug in clinical trials for the treatment of anemia in beta thalassemia and myelodysplastic syndromes. It is being developed by Acceleron Pharma in collaboration with Celgene.[2]

The FDA awarded orphan drug status in 2013 and fast track designation in 2015 for both indications.[3][4]

Clinical development[edit]

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

Structure[edit]

Luspatercept is a recombinant fusion protein derived from human activin receptor type IIb (ActRIIb) linked to a protein derived from immunoglobulin G.[7]

References[edit]

  1. ^ "Luspatercept". AdisInsight. Retrieved 15 February 2017.
  2. ^ josh@comocreative.com. "Luspatercept: Our Lead Product Candidate". Acceleronpharma.com. Retrieved 2017-05-22.
  3. ^ "FDA Fast Track Designation Granted to Luspatercept for the Treatment of Patients with Beta-Thalassemia". Business Wire. 2015-05-18. Retrieved 2017-05-22.
  4. ^ a b "Luspatercept". AdisInsight. Retrieved 1 February 2017.
  5. ^ "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 - Full Text View". ClinicalTrials.gov. Retrieved 2017-05-22.
  6. ^ "A Study of Luspatercept (ACE-536) to Treat Anemia Due to Very Low, Low, or Intermediate Risk Myelodysplastic Syndromes - Full Text View". ClinicalTrials.gov. Retrieved 2017-05-22.
  7. ^ "Luspatercept". NCI Thesaurus. National Cancer Institute.