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Luspatercept Injectable Product
Luspatercept is an erythroid maturation agent that binds to and inhibits activin A and related proteins, promoting late-stage erythroid differentiation and hemoglobin production.
Luspatercept is an erythroid maturation agent that binds to and inhibits activin A and related proteins, promoting late-stage erythroid differentiation and increasing hemoglobin production. Used for Beta-thalassemia dependent on regular transfusions, Myelodysplastic syndrome with ring sideroblasts.
At a glance
| Generic name | Luspatercept Injectable Product |
|---|---|
| Also known as | luspatercept |
| Sponsor | Sun Yat-sen University |
| Drug class | Erythroid maturation agent |
| Target | Activin A; TGF-β superfamily ligands |
| Modality | Small molecule |
| Therapeutic area | Hematology |
| Phase | FDA-approved |
Mechanism of action
Luspatercept acts as a ligand trap by binding activin A and other TGF-β superfamily members that normally suppress erythroid maturation. By sequestering these inhibitory signals, it allows erythroid progenitor cells to progress through terminal differentiation stages, increasing red blood cell production and hemoglobin levels. This mechanism is particularly effective in conditions characterized by ineffective erythropoiesis.
Approved indications
- Beta-thalassemia dependent on regular transfusions
- Myelodysplastic syndrome with ring sideroblasts
Common side effects
- Bone pain
- Arthralgia
- Headache
- Fatigue
- Dizziness
Key clinical trials
- MYELOMATCH: A Screening Study to Assign People With Myeloid Cancer to a Treatment Study or Standard of Care Treatment Within myeloMATCH (MyeloMATCH Screening Trial) (PHASE2)
- A Study of Elritercept to Treat Anemia in Adults With Very Low, Low, or Intermediate Risk Myelodysplastic Syndromes (MDS) (PHASE2)
- Luspatercept in Patients Affected With Rare Inherited Anemias (PHASE2)
- A Study to Evaluate Long-term Safety in Participants Who Have Participated in Other Luspatercept (ACE-536) Clinical Trials (PHASE3)
- Efficacy of Luspatercept in ESA-naive LR-MDS Patients With or Without Ring Sideroblasts Who do Not Require Transfusions (PHASE2)
- The Safety and Efficiency of Luspatercept in Chinese Adults With Transfusion Dependent β-thalassemia: a Real-world Study (PHASE4)
Primary sources
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |