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Drug

Luspatercept

Approved · EMA
Emerging researchLate-stage development

Also known as Reblozyl.

RxNorm2262544UNIIAQK7UBA1LS
4
Research papers
25
Active clinical trials
1
Regulatory events
Latest activity
beta

Recent clinical, regulatory, research and industry developments relating to this drug.

A Phase II, Open-label, Single Arm Study to Evaluate the Efficacy of Luspatercept in Erythropoiesis-stimulating Agent Naive Lower-risk MDS Patients With or Without Ring Sideroblasts Who do Not Require RBC Transfusions

Clinical trial2026-07-31Primary completion · ClinicalTrials.gov

Master Screening and Reassessment Protocol (MSRP) for the NCI MyeloMATCH Clinical Trials

Clinical trial2026-07-10Results expected Q2 2029 · ClinicalTrials.gov

A Phase 3b, Open-label, Single-arm, Rollover Study to Evaluate Long-term Safety in Subjects Who Have Participated in Other Luspatercept (ACE-536) Clinical Trials

Clinical trial2026-07-01Results expected Q2 2028 · ClinicalTrials.gov

A Phase 3, Open-label, Randomized Study to Compare the Efficacy and Safety of Luspatercept (ACE-536) vs Epoetin Alfa for the Treatment of Anemia Due to Revised International Prognostic Scoring System (IPSS-R) Very Low, Low, or Intermediate-Risk Myelodysplastic Syndrome (MDS) in Erythropoiesis-Stimulating Agent (ESA)-Naive Participants Who Are Non-Transfusion Dependent (NTD): The "ELEMENT-MDS" Trial

Clinical trial2026-06-30Results expected Q1 2028 · ClinicalTrials.gov

Safety and Efficacy Study of the Tapering Dose of Luspatercept in Patients With Lower-risk Myelodysplastic Syndromes

Clinical trial2026-06-30Primary completion · ClinicalTrials.gov

A Phase 2, Study for the Treatment of Anemia With Alpha (α)-Thalassemia to Determine the Efficacy and Safety of Luspatercept (BMS-986346/ACE-536) in Adults and Evaluate the Safety and Pharmacokinetics in Adolescents

Clinical trial2026-06-24Results expected Q3 2027 · ClinicalTrials.gov

A Pilot, Open-Label Study of Luspatercept for Patients With Lower Risk Myelodysplastic Syndromes (MDS)

Clinical trial2026-06-22Results expected Q2 2028 · ClinicalTrials.gov

Approval: Reblozyl (EMA)

Regulatory2020-06-25EMA

Luspatercept in Myelodysplastic Syndromes: Who and When?

Research2020-01-21Hematology/oncology clinics of North America

Luspatercept in Patients with Lower-Risk Myelodysplastic Syndromes.

Research2020-01-01The New England journal of medicine

Luspatercept improves hemoglobin levels and blood transfusion requirements in a study of patients with β-thalassemia.

Research2019-01-07Blood

Luspatercept for the treatment of anemia in myelodysplastic syndromes and primary myelofibrosis.

Research2019-01-02Blood

Profile

Identifiers & mechanism

Canonical identifiers, marketed brand names and mechanism, resolved across RxNorm, ChEMBL and ATC.

Canonical name
Luspatercept
Aliases & brands
Reblozyl
RxNorm CUI
2262544
UNII
AQK7UBA1LS
Regulatory jurisdictions
ema

Regulatory timeline

1 event

The complete regulatory record, grouped by authority — approvals, safety advisories and label changes. Each authority shows its most recent events; expand one to read its full history.

Earliest approval
2020-06-25
Latest approval
2020-06-25
Authorities
EMA
Total events
1
emaEuropean Medicines Agency· 1 event
2020-06-25Approval
Approval: Reblozyl (EMA)
Indication: Reblozyl is indicated in adults for the treatment of transfusion-dependent anaemia due to very low, low and intermediate-risk myelodysplastic syndromes (MDS).Reblozyl is indicated… Show full indication

Reblozyl is indicated in adults for the treatment of transfusion-dependent anaemia due to very low, low and intermediate-risk myelodysplastic syndromes (MDS).Reblozyl is indicated in adults for the treatment of anaemia associated with transfusion dependent and non transfusion dependent beta thalassaemia.  Reblozyl is indicated in adults for the treatment of transfusion-dependent anaemia due to very low, low and intermediate-risk myelodysplastic syndromes (MDS).Reblozyl is indicated in adults for the treatment of anaemia associated with transfusion dependent and non transfusion dependent beta thalassaemia. 

Evidence ↗

Contains information from the European Medicines Agency (European Medicines Agency), © EMA, reused under CC BY 4.0.

Clinical trials

42 trials

The current development programme across all trial phases — status mix, phase distribution and the late-stage studies shaping the evidence base.

Development programme
CLINICALTRIALS.GOV · LIVE REGISTRY
42
registered trials across all phases
LATEST COMPLETION 2026
25
Active studies
17
Recruiting
16
Late-stage (III+)
12
Completed
5
Discontinued
PHASE DISTRIBUTIONn = 42
Phase 1 / 23Phase 223Phase 2 / 32Phase 38Phase 43Phase N / A3

Late-stage studies

Phase III+ trials still open or recently active — where late-stage evidence is being generated.

A Phase 4 Study to Evaluate Safety and Effectiveness of Luspatercept (ACE-536) for the Treatment of Anemia Due to IPSS-R Very Low, Low, or Intermediate Risk Myelodysplastic Syndromes (MDS) With Ring Sideroblasts Who Require Red Blood Cell Transfusions in Participants Who Have Had Unsatisfactory Response to or Are Ineligible to Erythropoietin Based Therapy and in Participants With Transfusion Dependent Anemia Due to Beta-Thalassemia

Active, not recruitingPH IVBristol-Myers Squibbn=852028

A Randomized, Controlled Trial Comparing the Safety and Efficacy of Luspatercept Plus Cyclosporine Versus Cyclosporine Alone for the Treatment of Newly Diagnosed Non-transfusion-dependent Non-severe Aplastic Anemia (NSAA)

WithdrawnPH IVPeking Union Medical College Hospital2025

Roxadustat Combined With Luspatercept Versus Luspatercept Monotherapy in the Treatment of Refractory Myelodysplastic Syndrome With Ring Sideroblasts (MDS-RS): A Prospective Randomized Controlled Study

Unknown statusPH IVPeking Union Medical College Hospitaln=622025

A Phase 3, Open-label, Randomized Study to Compare the Efficacy and Safety of Luspatercept (ACE-536) vs Epoetin Alfa for the Treatment of Anemia Due to Revised International Prognostic Scoring System (IPSS-R) Very Low, Low, or Intermediate-Risk Myelodysplastic Syndrome (MDS) in Erythropoiesis-Stimulating Agent (ESA)-Naive Participants Who Are Non-Transfusion Dependent (NTD): The "ELEMENT-MDS" Trial

Active, not recruitingPH IIIBristol-Myers Squibbn=4022031

The Efficacy and Safety of Luspatercept in Preventing Poor Erythroid Engraftment After Allo-HSCT for Hematological Malignancies With Moderate to Severe Myelofibrosis: A Prospective, Multicenter, Randomized Controlled Study

Not yet recruitingPH II/IIINanfang Hospital, Southern Medical Universityn=1962030

Recent completions

Trials that read out recently, adding to the completed evidence base.

A Phase 2, Multicenter, Single-Arm Bridging Study to Evaluate the Efficacy, Pharmacokinetics, and Safety of Luspatercept (ACE-536) for the Treatment of Anemia Due to IPSS-R Very Low, Low or Intermediate Risk Myelodysplastic Syndromes(MDS) in Chinese and Japanese Subjects With Ring Sideroblasts Who Require Red Blood Cell Transfusions

CompletedPH IICelgenen=302026

A Randomized, Case Controlled Clinical Trial Evaluating the Efficiency and Safety of Luspatercept Plus Cyclosporine Versus Cyclosporine in Newly Diagnosed Transfusion Independent Non-severe Aplastic Anemia (NSAA).

CompletedPH IIBing Hann=582025

A Phase 2, Multicenter, Single-arm Study to Evaluate the Efficacy, Pharmacokinetics, and Safety of Luspatercept (ACE-536) for the Treatment of Anemia Due to IPSS-R Very Low, Low or Intermediate Risk Myelodysplastic Syndromes (MDS) in Japanese Subjects Who Are Not Requiring Red Blood Cell Transfusion

CompletedPH IICelgenen=212023

A Phase 2, Double-Blind, Randomized, Placebo-Controlled Multicenter Study to Determine the Efficacy and Safety of Luspatercept (ACE-536) Versus Placebo in Adults With Non-Transfusion Dependent Beta (β)-Thalassemia (The BEYOND™ Study)

CompletedPH IICelgenen=1452022

Luspatercept in Metastatic Adult Granulosa Cell Tumor (AGCT) of the Ovary

CompletedN/AUniversity Health Network, Toronton=12022

A Phase-2 Study To Determine Efficacy and Safety of Luspatercept in Subjects With Myeloproliferative Neoplasm-Associated Myelofibrosis and Anemia With or Without Red Blood Cell-Transfusion Dependence

CompletedPH IICelgenen=952022

Research activity

4 papers

Key research shaping understanding of this drug, combining the latest publications with the most influential evidence.

Publications over time
20192020
Major research themes1
Erythrocyte Transfusion1
Journals, researchers & institutions
Top journals
  • Blood2
  • Hematology/oncology clinics of North America1
  • The New England journal of medicine1
Leading researchers
  • Fenaux P2
  • Komrokji RS2
  • Laadem A2
  • Platzbecker U2
  • Sherman ML2
  • Adès L1
  • Arrizabalaga B1
  • Attie KM1
Leading institutions
free-text, unnormalised
  • Assistance Publique-Hôpitaux de Paris and Université Paris 71
  • Cardarelli Hospital1
  • Centre d'Investigations Cliniques1
  • Department of Clinical and Biological Sciences1
  • Laiko General Hospital1
  • Medical Clinic and.1
References & data sources
  • RxNorm (U.S. National Library of Medicine) — drug identity
  • Europe PMC — research literature
  • ClinicalTrials.gov — clinical trials
  • Regulatory event sources are credited in the Regulatory Timeline above.