Luspatercept
Luspatercept is a recombinant fusion protein used to treat certain types of anemia by improving red blood cell production. It works by binding TGF-β superfamily ligands and reducing Smad2/3 signaling, which helps restore normal erythropoiesis. The medication is primarily used in patients with beta-thalassemia and certain myelodysplastic syndromes.
Luspatercept is an anemia treatment that modulates TGF-β signaling to improve red blood cell production in patients with beta-thalassemia and myelodysplastic syndromes.
Key facts at a glance
- ATC
- B03XA06
- RxNorm
- 2262544
- Generic
- Luspatercept
- Class
- atc-b03
- Brands
- Reblozyl
- Availability
- rx
- Lactation
- unknown
- Routes
- —
- Forms
- injection
- Regions
- US, EU, CN, JP
- Monitoring
- —
This page is for informational purposes only.
Do not use this information to diagnose yourself or to replace a physician's advice. Always consult a qualified healthcare provider before starting, stopping, or changing any medication. If you believe you are experiencing a medical emergency, call your local emergency number immediately.
Luspatercept is indicated for the treatment of anemia in adult patients with beta-thalassemia who require regular red blood cell transfusions, as well as in patients with very low to intermediate-risk myelodysplastic syndromes with ring sideroblasts who have not responded to or are ineligible for erythropoiesis-stimulating agents.
Luspatercept is a recombinant fusion protein that binds to TGF-β superfamily ligands, thereby diminishing Smad2/3 signaling. By modulating this pathway, it reduces abnormal inhibition of erythropoiesis and promotes the maturation of red blood cell precursors, leading to improved hemoglobin levels and reduced transfusion burden.
Luspatercept is administered as a subcutaneous injection, typically every 3 weeks. Common side effects may include bone pain, fatigue, and headache. Patients should be monitored for hypertension, thrombosis, and potential disease progression in MDS. It should not be used during pregnancy and requires appropriate contraception during treatment.
Official drug label
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This information is for educational purposes only and is not a substitute for professional medical advice, diagnosis, or treatment.
This content is for general informational and educational purposes only and is not a substitute for professional medical advice, diagnosis, or treatment. Always seek the guidance of a qualified healthcare provider with any questions you have about a medical condition, and never disregard professional medical advice or delay seeking it because of something you read here.
If you think you may have a medical emergency, call your local emergency number or go to the nearest emergency department immediately.
Medically reviewed by WellAlly Editorial Team Last reviewed July 26, 2026. · Read our editorial policy