luspatercept
Orphan Drug Cold Chain RequiredFDA Approved, EMA Approved
Description
Luspatercept is a recombinant fusion protein that acts as an erythroid maturation agent by binding to select TGF-beta superfamily ligands. It promotes late-stage erythropoiesis and reduces transfusion burden in patients with beta-thalassemia including hemoglobin E-beta-thalassemia syndrome. The drug enhances red blood cell maturation and production.
Indications & Therapeutic Use
beta-thalassemia, hemoglobin E-beta-thalassemia syndrome, myelodysplastic syndromes with ring sideroblasts
Global Availability (8 countries)
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Available Through
This drug is available for procurement through the following fulfilment partner.
Fulfilment Partner
luspatercept
| Generic Name | luspatercept |
| Brands | 1 brand available |
| Active Ingredient | luspatercept-aamt |
| Drug Class | beta-thalassemia |
| Manufacturer | Bristol Myers Squibb/Celgene |
| Dosage Forms | subcutaneous injection, 25 mg/vial, 75 mg/vial powder for solution |
| Medical Code | B03XA05 |
| Orphan Status | Yes — Orphan Drug |
| Cold Chain | Required |
| Lead Time | 10 days |
| Reg. Status | FDA Approved, EMA Approved |
| Clinical Trial | NCT02604433 |
| Countries | 8 countries |
Authority Signals
Last Verified3/21/2026
Data ConfidenceHigh (AI + Pharmacist)
Source Citations9 Validated Nodes