Reblozyl

(Luspatercept)

ApprovedProtein / enzyme

Luspatercept is used to treat anemia (low red blood cell counts) in patients with certain blood disorders, including myelodysplastic syndromes and beta-thalassemia, by helping the body produce more red blood cells.[2] Reblozyl is currently approved and in use across 16 indications[4].[1]

Reblozyl is one of 320 small molecule programs targeting Ovarian Granulosa Cell Tumor. It is currently in Approved.

Indications

Total Trials

Active Trials

319

Competitors

FDA Application

NDA/BLA/ANDA regulatory filings

Pipeline

Status by Indication[4][1]

Indication

Ovarian Granulosa Cell TumorActive, not recruiting

Maligant Granulosa Cell Tumor of OvaryActive, not recruiting

Anemia (phenotype)Unknown

AnemiaActive, not recruiting

MyelofibrosisCompleted

Myelodysplastic SyndromeActive, not recruiting

Myelodysplastic SyndromesActive, not recruiting

Immune ThrombocytopeniaActive, not recruiting

MDSRecruiting

AINRecruiting

Ovarian Granulosa Cell TumorPreclinical

Active, not recruiting

Maligant Granulosa Cell Tumor of OvaryPreclinical

Active, not recruiting

Anemia (phenotype)Preclinical

Unknown

AnemiaApproved

Active, not recruiting

MyelofibrosisPhase 2

Completed

Myelodysplastic SyndromePhase 3

Active, not recruiting

Myelodysplastic SyndromesPhase 3

Active, not recruiting

Immune ThrombocytopeniaPreclinical

Active, not recruiting

MDSPreclinical

Recruiting

AINPhase 2

Recruiting

Showing 10 of 16 indications

Competition

Competitive Landscape

76 competitors in Newly Diagnosed

#DrugCompanyPhaseTrials

Venclexta

AbbVie

Phase 3

15 2

Cytosar

Teva

Phase 3

14 3

Rituxan

Roche

Phase 3

11 4

Reblozyl

Bristol-Myers Squibb

Approved

1 5

Oncovin

Eli Lilly

Phase 3

7 6

Adriamycin

Pfizer

Phase 3

6 7

Darzalex

Johnson & Johnson

Phase 3

4 8

Decadron

AAspen Global Incorporated

Phase 3

14 9

Vanflyta

Daiichi Sankyo

Phase 3

3 10

Oncovin

Eli Lilly

Phase 3

Ranked by pipeline maturity and clinical activity. Current drug highlighted in teal.

Timeline

Expected Milestones

Q4 2026

Phase 2Recruiting

Myelofibrosis

NCT06517875

Active, not recruiting

Lower-Risk Myelodysplastic Syndromes (TD LR-MDS)

NCT07465029

Active, not recruiting

Thalassemia

NCT06596642

Q1 2027

Phase 3Not yet recruiting

Monotherapy

NCT06927232

Phase 3Active, not recruiting

Myelodysplastic Syndrome

NCT05181592

Q2 2027

PreclinicalRecruiting

Anemia

NCT07663864

Clinical

Trials by Indication[1]

Myelodysplastic Syndromes15 trials

A Study to Evaluate Luspatercept Treatment Patterns and Outcomes in Erythropoiesis-Stimulating Agents-Naïve Patients With Lower-Risk Myelodysplastic Syndromes in the United States

Completed· Bristol-Myers Squibb logo

Bristol-Myers Squibb··NCT06851065·418 enrolled

A Study of Luspatercept (ACE-536) to Treat Anemia Due to Very Low, Low, or Intermediate Risk Myelodysplastic Syndromes

Completed· Bristol-Myers Squibb logo

Bristol-Myers Squibb·Phase 3·NCT02631070·229 enrolled

Extension Study to Evaluate Long-Term Effects of Luspatercept in Patients With Myelodysplastic Syndromes (MDS) (A536-05/MK-6143-003)

Completed· Merck & Co. logo

Merck & Co.·Phase 2·NCT02268383·75 enrolled

Show 12 more trials

A Study to Evaluate Treatment Patterns, Outcomes, and Healthcare Resource Utilization in Patients With Lower-Risk Myelodysplastic Syndromes Treated With Luspatercept

Completed· Bristol-Myers Squibb logo

Bristol-Myers Squibb··NCT07075406·250 enrolled

The Tapering Dose of Luspatercept in Patients With Lower-risk Myelodysplastic Syndromes

Recruiting·IInstitute of Hematology & Blood Diseases Hospital, China·Phase 2·NCT05925504·36 enrolled

A Study to Evaluate the Efficacy, Drug Levels and Safety of Luspatercept (ACE-536) for the Treatment of Anemia Due to IPSS-R Very Low, Low, or Intermediate Risk Myelodysplastic Syndromes in Chinese and Japanese Participants With Ring Sideroblasts Who Require Red Blood Cell Transfusions

Unknown status· Bristol-Myers Squibb logo

Bristol-Myers Squibb·Phase 2·NCT04477850·30 enrolled

A Retrospective Study to Describe Real-World Treatment Patterns and Clinical Outcomes Among Patients With Myelodysplastic Syndromes

Withdrawn· Bristol-Myers Squibb logo

Bristol-Myers Squibb··NCT06581055

An Efficacy and Safety Study 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

Completed· Bristol-Myers Squibb logo

Bristol-Myers Squibb·Phase 2·NCT03900715·21 enrolled

Treatment Patterns and Outcomes in Patients With Lower-risk Myelodysplastic Syndromes Treated With Luspatercept in China

Active, not recruiting· Bristol-Myers Squibb logo

Bristol-Myers Squibb··NCT07073690·80 enrolled

A Study to Compare the Efficacy and Safety of Luspatercept (ACE-536) Versus Epoetin Alfa for the Treatment of Anemia Due to IPSS-R Very Low, Low, or Intermediate Risk Myelodysplastic Syndromes (MDS) Participants Who Require Red Blood Cell Transfusions and Are ESA Naïve

Active, not recruiting· Bristol-Myers Squibb logo

Bristol-Myers Squibb·Phase 3·NCT03682536·363 enrolled

Efficacy and Safety of Maximum Labeled Dose Luspatercept in Lower-Risk Myelodysplastic Syndromes

Not yet recruiting·IInstitute of Hematology & Blood Diseases Hospital, China·Phase 3·NCT07894861·75 enrolled

A Multicenter, Phase Ib/II Study That Combines Luspatercept and Lenalidomide (L2) in Lower-risk, Non-del(5q) MDS Patients

Suspended·MMikkael Sekeres, MD·Preclinical·NCT04539236·50 enrolled

Real World Use of Luspatercept in Lower Risk Myelodysplastic Syndromes and Beta-thalassemia Patients

Terminated· Bristol-Myers Squibb logo

Bristol-Myers Squibb··NCT07681440·30 enrolled

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 recruiting· Bristol-Myers Squibb logo

Bristol-Myers Squibb·Phase 3·NCT05949684·402 enrolled

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

Active, not recruiting·MM.D. Anderson Cancer Center·Phase 2·NCT06113302·10 enrolled

Thalassemia8 trials

The Safety and Efficiency of Luspatercept in Chinese Adults With Transfusion Dependent β-thalassemia: a Real-world Study

Unknown status·SSun Yat-sen University·Phase 4·NCT05462548·20 enrolled

Clinical Observation of Luspatercept in Treatment of Chinese Adult β-thalassaemia Patients With TD β-thalassemia

Unknown status·HHematology department of the 920th hospital·N/A·NCT06164821·10 enrolled

A Study to Evaluate Luspatercept (ACE-536) in Chinese Participants Who Require Regular Red Blood Cell Transfusions Due to Beta (β)-Thalassemia.

Active, not recruiting· Bristol-Myers Squibb logo

Bristol-Myers Squibb·Phase 2·NCT05567458·94 enrolled

Show 5 more trials

Extension Study to Evaluate the Safety and Efficacy of Luspatercept in Participants With β-Thalassemia Previously Enrolled in A536-04 (A536-06/MK-6143-004)

Completed· Merck & Co. logo

Merck & Co.·Phase 2·NCT02268409·51 enrolled

A Real-World Study of β-Thalassemia Major Treatment With Luspatercept in Taiwan

Active, not recruiting· Bristol-Myers Squibb logo

Bristol-Myers Squibb··NCT06596642·61 enrolled

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

Completed· Bristol-Myers Squibb logo

Bristol-Myers Squibb·Phase 3·NCT02604433·336 enrolled

A Study to Determine the Efficacy and Safety of Luspatercept in Adults With Non Transfusion Dependent Beta (β)-Thalassemia

Completed· Bristol-Myers Squibb logo

Bristol-Myers Squibb·Phase 2·NCT03342404·145 enrolled

REal-World Application of Luspatercept in Adults With Transfusion-Dependent Beta-Thalassemia in the Middle East (RELATE): A Non-interventional Retrospective and Prospective Observational Study

Recruiting· Bristol-Myers Squibb logo

Bristol-Myers Squibb··NCT07215975·200 enrolled

Anemia8 trials

Luspatercept for Anemia in Lower Risk MDS or Non-proliferative MDS/MPN Neoplasms

Recruiting·HH. Lee Moffitt Cancer Center and Research Institute·Phase 2·NCT05732961·70 enrolled

The Efficacy and Safety of Luspatercept in Improving Early Anemia After HSCT

Not yet recruiting·IInstitute of Hematology & Blood Diseases Hospital, China·Phase 2·NCT07450313·20 enrolled

Luspatercept for the Treatment of Anemia Following Allogeneic Hematopoietic Stem Cell Transplantation(Allo-HSCT)

Recruiting·ZZhujiang Hospital·Phase 1/2·NCT07362095·46 enrolled

Show 5 more trials

Efficacy and Safety of Luspatercept for the Treatment of Anemia Due to MDS With del5q, Refractory/Resistant/Intolerant to Prior Treatments, RBC-TD

Recruiting·AAssociazione Qol-one·Phase 2·NCT05924100·22 enrolled

A Multicenter Phase 2 Study of Luspatercept in Patients With Anemia Due to Chronic Kidney Disease

Recruiting·MMemorial Sloan Kettering Cancer Center·Phase 2·NCT07725497·30 enrolled

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

Active, not recruiting· Bristol-Myers Squibb logo

Bristol-Myers Squibb·Phase 2·NCT05664737·189 enrolled

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 recruiting· Bristol-Myers Squibb logo

Bristol-Myers Squibb·Approved·NCT05891249·85 enrolled

Efficacy and Safety of Luspatercept in Treating Chemotherapy-inducing Anemia in Acute Myeloid Leukemia: a Multicenter, Prospective, Single-arm Study

Recruiting·GGuangdong Second Provincial General Hospital·Preclinical·NCT07663864·40 enrolled

Myelodysplastic Syndrome5 trials

A Post-Marketing Surveillance Study to Assess Safety of Luspatercept in Korean Patients With Myelodysplastic Syndrome or β-thalassemia

Recruiting· Bristol-Myers Squibb logo

Bristol-Myers Squibb··NCT06073860·104 enrolled

A Study to Assess Luspatercept in Lower-risk Myelodysplastic Syndrome Participants

Active, not recruiting· Bristol-Myers Squibb logo

Bristol-Myers Squibb·Phase 3·NCT06045689·106 enrolled

Study of Luspatercept for the Treatment of Anemia in Patients With Myelodysplastic Syndrome (MDS) (MK-6143-001)

Completed· Merck & Co. logo

Merck & Co.·Phase 2·NCT01749514·116 enrolled

Show 2 more trials

Assessment of Effectiveness and Safety of Luspatercept in Patients Suffering From Lower-risk Myelodysplastic Syndrome.

Active, not recruiting·GGWT-TUD GmbH·Phase 3·NCT05181592·70 enrolled

A Phase II Study of Combination Therapy With Luspatercept in Lower Risk Myelodysplasia: A Tier 1 MyeloMATCH Substudy

Not yet recruiting·NNational Cancer Institute (NCI)·Phase 2·NCT07463820·270 enrolled

Severe Aplastic Anemia2 trials

Treatment of Transfusion-dependent Nonsevere Aplastic Anemia With Luspatercept: a Multicenter Prospective Clinical Study

Not yet recruiting·TThe First Affiliated Hospital of Zhejiang Chinese Medical University·N/A·NCT06964971·90 enrolled

Efficacy and Safety in Transfusion Independent Non-severe Aplastic Anemia

Completed·BBing Han·Phase 2·NCT05399732·58 enrolled

19 more indications

Myelofibrosis2 trials

A Safety and Efficacy Study to Evaluate Luspatercept in Subjects With Myeloproliferative Neoplasm-associated Myelofibrosis Who Have Anemia With and Without Red Blood Cell-transfusion Dependence

Completed· Bristol-Myers Squibb logo

Bristol-Myers Squibb·Phase 2·NCT03194542·95 enrolled

Study of Momelotinib in Combination With Luspatercept in Participants With Transfusion Dependent Myelofibrosis

Recruiting· GSK logo

GSK·Phase 2·NCT06517875·56 enrolled

Monotherapy2 trials

Roxadustat Combined With Luspatercept Versus Luspatercept Monotherapy in the Treatment of Refractory MDS-RS

Unknown status·PPeking Union Medical College Hospital·Phase 4·NCT06006949·62 enrolled

AZA+Lus VS AZA Monotherapy in HR-MDS

Not yet recruiting·PPeking Union Medical College Hospital·Phase 3·NCT06927232·86 enrolled

Safety and Efficacy2 trials

Study to Evaluate the Long- Term Safety and Efficacy of Luspatercept in Subjects Who Received at Least One Dose of Luspatercept in the Compassionate Use Phase

Active, not recruiting·FFondazione per la Ricerca sulle Anemie ed Emoglobinopatie in Italia··NCT06913634·140 enrolled

To Evaluate Long- Term Safety and Efficacy of Luspatercept

Active, not recruiting·FFondazione per la Ricerca sulle Anemie ed Emoglobinopatie in Italia··NCT06875219·350 enrolled

Myelodysplastic Syndromes (MDS)2 trials

Treatment Patterns and Effectiveness of Luspatercept in the Real World

Completed· Bristol-Myers Squibb logo

Bristol-Myers Squibb··NCT06971185·430 enrolled

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

Recruiting· Bristol-Myers Squibb logo

Bristol-Myers Squibb·Phase 3·NCT04064060·665 enrolled

Newly Diagnosed1 trial

Luspatercept Plus CsA vs CsA for the Treatment of Newly Diagnosed Non-Transfusion-Dependent NSAA

Not yet recruiting·PPeking Union Medical College Hospital·Phase 4·NCT06424639·58 enrolled

Efficacy1 trial

Efficacy of Luspatercept in ESA-naive LR-MDS Patients With or Without Ring Sideroblasts Who do Not Require Transfusions

Recruiting·UUniversity of Leipzig·Phase 2·NCT05384691·213 enrolled

Beta-Thalassemia1 trial

Study to Evaluate the Safety and Efficacy of Luspatercept (ACE-536) in Participants With Beta-thalassemia (A536-04/MK-6143-002)

Completed· Merck & Co. logo

Merck & Co.·Phase 2·NCT01749540·64 enrolled

Metastatic1 trial

Luspatercept in Metastatic AGCT of the Ovary

Completed·UUniversity Health Network, Toronto·N/A·NCT06254781·1 enrolled

Efficacy and Safety1 trial

Efficacy and Safety of Luspatercept: a Study by Fondazione Italiana Sindromi Mielodisplastiche

Completed·FFondazione Italiana Sindromi Mielodisplastiche-ETS··NCT05520749·215 enrolled

Myelodysplastic/Myeloproliferative Neoplasm1 trial

Luspatercept With or Without Hydroxyurea for the Treatment of Myelodysplastic/Myeloproliferative Neoplasms With Ring Sideroblasts and Thrombocytosis or Unclassifiable With Ring Sideroblasts

Terminated·MMayo Clinic·Phase 2·NCT05005182·3 enrolled

AIN1 trial

Luspatercept for Clonal Cytopenias of Uncertain Significance

Recruiting·WWeill Medical College of Cornell University·Phase 2·NCT06788691·50 enrolled

Immune Thrombocytopenia1 trial

Prospective Evaluation of Diagnosis and Treatment of Patients With Autoimmune Cytopenias Including Autoimmune Hemolytic Anemia, Immune Thrombocytopenia, and Chronic Idiopathic/Autoimmune Neutropenia

Recruiting·FFondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico··NCT05931718·200 enrolled

Myeloid1 trial

Connect® Myeloid Disease Registry

Terminated· Bristol-Myers Squibb logo

Bristol-Myers Squibb··NCT01688011·2,013 enrolled

Acute Myeloid Leukemia1 trial

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

Recruiting·NNational Cancer Institute (NCI)·Phase 2·NCT05564390·2,000 enrolled

Lower-risk Myelodysplastic Syndromes (TD LR-MDS)1 trial

Observational Retrospective Study on Incidence, First-line Treatment Patterns, and Clinical Outcomes in Transfusion-dependent Lower-risk Myelodysplastic Syndromes in Spain Using the BIG-PAC® Database

Active, not recruiting· Bristol-Myers Squibb logo

Bristol-Myers Squibb··NCT07465029·1,300 enrolled

MDS1 trial

A Randomized Phase I/ II Multicenter Study Evaluating Combination of Luspatercept in LR-MDS Without RS Having Failed or Being Ineligible to ESA

Recruiting·GGroupe Francophone des Myelodysplasies·Preclinical·NCT05181735·150 enrolled

Myelodysplasia1 trial

Plasmatic Biomarkers Associated With Short-term Luspatercept Treatment of Lower Risk Myelodysplastic Syndromes (MDS) Patients

Not yet recruiting·UUniversity Hospital, Grenoble·Approved·NCT07830004·150 enrolled

MDS (Myelodysplastic Syndrome)1 trial

A Phase II Study of Luspatercept Plus Darbepoetin Alfa in Non-mutated SF3B1 Lower-risk Myelodysplastic Syndromes

Recruiting·YYale University·Phase 2·NCT07096297·60 enrolled

Myelodysplastic Neoplasms1 trial

A Prospective, Multicenter, Non-Interventional Study Assessing First-line Luspatercept in Anemic Patients With Very Low, Low, or Intermediate Risk Myelodysplastic Neoplasms Requiring Red Blood Cell Transfusions

Not yet recruiting· Bristol-Myers Squibb logo

Bristol-Myers Squibb··NCT07752121·190 enrolled

Regulatory

FDA Approval Timeline[5]

Nov 8, 2019

REBLOZYL

BLA BLA761136

Sources

References

[1]
ClinicalTrials.gov — 61 trial records for Luspatercept. U.S. National Library of Medicine, National Institutes of Health.
[2]
MedlinePlus Drug Information — Reblozyl drug information. U.S. National Library of Medicine, National Institutes of Health.
[3]
ChEMBL — Luspatercept compound record. European Bioinformatics Institute (EMBL-EBI).
[4]
Open Targets Platform — Drug–disease associations for Luspatercept. EMBL-EBI & Wellcome Sanger Institute.
[5]
OpenFDA — FDA approval records (BLA BLA761136). U.S. Food & Drug Administration.
[6]
SEC EDGAR — Bristol-Myers Squibb company filings. U.S. Securities and Exchange Commission.

Details

Drug Info

Quick Facts

Mechanism of Action: Transforming Growth Factor Beta Inhibitor[3]
Modality: Protein / enzyme[3]
Highest Phase: Approved[1]
Company: Bristol-Myers Squibb

Reblozyl

Status by Indication[4][1]

Competitive Landscape

Expected Milestones

Q4 2026

Q1 2027

Q2 2027

Trials by Indication[1]

Myelodysplastic Syndromes15 trials

Thalassemia8 trials

Anemia8 trials

Myelodysplastic Syndrome5 trials

Severe Aplastic Anemia2 trials

Myelofibrosis2 trials

Monotherapy2 trials

Safety and Efficacy2 trials

Myelodysplastic Syndromes (MDS)2 trials

Newly Diagnosed1 trial

Efficacy1 trial

Beta-Thalassemia1 trial

Metastatic1 trial

Efficacy and Safety1 trial

Myelodysplastic/Myeloproliferative Neoplasm1 trial

AIN1 trial

Immune Thrombocytopenia1 trial

Myeloid1 trial

Acute Myeloid Leukemia1 trial

Lower-risk Myelodysplastic Syndromes (TD LR-MDS)1 trial

MDS1 trial

Myelodysplasia1 trial

MDS (Myelodysplastic Syndrome)1 trial

Myelodysplastic Neoplasms1 trial

FDA Approval Timeline[5]

References

Drug Info

Quick Facts

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