Luspatercept Explained

Pronounce:
Tradename:Reblozyl
Dailymedid:Luspatercept
Pregnancy Au:D
Pregnancy Au Comment:[1]
Routes Of Administration:Subcutaneous injection
Atc Prefix:B03
Atc Suffix:XA06
Legal Au:S4
Legal Au Comment:[2]
Legal Ca:Rx-only
Legal Ca Comment:/ Schedule D[3] [4]
Legal Us:Rx-only
Legal Us Comment:[5]
Legal Eu:Rx-only
Legal Eu Comment:[6]
Cas Number:1373715-00-4
Pubchem:347911311
Drugbank:DB12281
Chemspiderid:none
Unii:AQK7UBA1LS
Kegg:D11701
Synonyms:ACE-536, luspatercept-aamt
C:3350
H:5070
N:906
O:1044
S:38

Luspatercept, sold under the brand name Reblozyl, is a medication used for the treatment of anemia in beta thalassemia and myelodysplastic syndromes.

The US Food and Drug Administration (FDA) considers it to be a first-in-class medication.[7]

Medical uses

Luspatercept is indicated for the treatment of adults with transfusion-dependent anemia due to very low, low and intermediate-risk myelodysplastic syndromes (MDS) with ring sideroblasts, who had an unsatisfactory response to or are ineligible for erythropoietin-based therapy.

Luspatercept is indicated for the treatment of adults with transfusion-dependent anaemia associated with beta thalassaemia.

Side effects

Possible adverse effects include temporary bone pain, joint pains (arthralgias), dizziness, elevated blood pressure (hypertension) and elevated uric acid levels (hyperuricemia). There was also an increased risk of thrombosis (blood clots) in patients who have risk factors for thrombosis who are taking luspatercept.[8]

Structure and mechanism

Luspatercept is a recombinant fusion protein derived from human activin receptor type IIb (ActRIIb) linked to a protein derived from immunoglobulin G.[9] It binds TGF (transforming growth factor beta) superfamily ligands to reduce SMAD signaling. The reduction in SMAD signaling leads to enhanced erythroid maturation.

History

Phase III trials evaluated the efficacy of luspatercept for the treatment of anemia in the hematological disorders beta thalassemia[10] and myelodysplastic syndromes.[11]

It was developed by Acceleron Pharma in collaboration with Celgene.[12]

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.[13] [14] [15] [16] Luspatercept was approved for medical use in the European Union in June 2020.[17]

The U.S. Food and Drug Administration (FDA) awarded orphan drug status in 2013, and fast track designation in 2015.[18]

Research

Luspatercept is being evaluated for use in adults with non-transfusion dependent beta thalassemia.[19]

Notes and References

  1. Web site: Reblozyl . Therapeutic Goods Administration (TGA) . 7 September 2021 . 17 September 2021.
  2. Web site: AusPAR: Luspatercept . Therapeutic Goods Administration (TGA) . 28 April 2022 . 28 April 2022.
  3. Web site: Summary Basis of Decision (SBD) for Reblozyl . Health Canada . 23 October 2014 . 29 May 2022.
  4. Web site: Health product highlights 2021: Annexes of products approved in 2021 . . 3 August 2022 . 25 March 2024.
  5. Web site: Reblozyl- luspatercept injection, powder, lyophilized, for solution . DailyMed . 17 September 2021.
  6. Web site: Reblozyl Product information . Union Register of medicinal products . 3 March 2023.
  7. Web site: New Drug Therapy Approvals 2019 . U.S. Food and Drug Administration . 31 December 2019 . 15 September 2020.
  8. Taher AT, Musallam KM, Cappellini MD . β-Thalassemias . The New England Journal of Medicine . 384 . 8 . 727–743 . February 2021 . 33626255 . 10.1056/NEJMra2021838 . 232049825 .
  9. Web site: Luspatercept . NCI Thesaurus . National Cancer Institute.
  10. Web site: A Phase 3, Double-Blind, Placebo Controlled Multicenter Study to Determine the Efficacy and Safety of Luspatercept (ACE-536) in Adults With Transfusion Dependent Beta (B)-Thalassemia . clinicaltrials.gov . 5 May 2022 . 21 January 2022.
  11. Web site: A Phase 3, Double-blind, Randomized Study to Compare the Efficacy and Safety of Luspatercept (ACE-536) Versus Placebo for the Treatment of Anemia Due to the IPSS-R Very Low, Low, or Intermediate Risk Myelodysplastic Syndromes in Subjects With Ring Sideroblasts Who Require Red Blood Cell Transfusions. . clinicaltrials.gov . 5 May 2022 . 19 November 2021.
  12. Web site: Luspatercept: Our Lead Product Candidate . Acceleron Pharma . 22 May 2017 . 5 March 2017 . https://web.archive.org/web/20170305123310/http://www.acceleronpharma.com/products/luspatercept/ . dead .
  13. FDA approves first therapy to treat patients with rare blood disorder . U.S. Food and Drug Administration (FDA) . 8 November 2019 . https://web.archive.org/web/20191113231630/https://www.fda.gov/news-events/press-announcements/fda-approves-first-therapy-treat-patients-rare-blood-disorder . 13 November 2019 . live . 13 November 2019.
  14. Web site: Reblozyl (luspatercept-aamt) FDA Approval History . Drugs.com . https://web.archive.org/web/20191113233045/https://www.drugs.com/history/reblozyl.html . 13 November 2019 . live . 13 November 2019.
  15. FDA Approves Reblozyl (luspatercept-aamt) for the Treatment of Anemia in Adults With Beta Thalassemia Who Require Regular Red Blood Cell Transfusions . Celgene . 8 November 2019 . https://web.archive.org/web/20191113233542/https://ir.celgene.com/press-releases/press-release-details/2019/FDA-Approves-REBLOZYL-luspatercept-aamt-for-the-Treatment-of-Anemia-in-Adults-With-Beta-Thalassemia-Who-Require-Regular-Red-Blood-Cell-Transfusions/default.aspx . 13 November 2019 . live . 13 November 2019.
  16. Web site: Drug Trials Snapshots: Reblozyl . U.S. Food and Drug Administration (FDA) . 8 November 2019 . 26 January 2020.
  17. Web site: Reblozyl EPAR . European Medicines Agency (EMA) . 28 April 2020 . 26 September 2020. Text was copied from this source which is copyright European Medicines Agency. Reproduction is authorized provided the source is acknowledged.
  18. FDA Fast Track Designation Granted to Luspatercept for the Treatment of Patients with Beta-Thalassemia . Acceleron/Celgene. Business Wire . 18 May 2015 . 22 May 2017.
  19. Piga A, Perrotta S, Gamberini MR, Voskaridou E, Melpignano A, Filosa A, Caruso V, Pietrangelo A, Longo F, Tartaglione I, Borgna-Pignatti C, Zhang X, Laadem A, Sherman ML, Attie KM . 6 . Luspatercept improves hemoglobin levels and blood transfusion requirements in a study of patients with β-thalassemia . Blood . 133 . 12 . 1279–1289 . March 2019 . 30617198 . 10.1182/blood-2018-10-879247 . 6440118 . free .

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