Luspatercept

Erythroid Maturation Agent — BLOOD AND BLOOD FORMING ORGANS

Key facts

Luspatercept is a drug in the Erythroid Maturation Agent class catalogued in DrugCentral. It was first approved by the FDA in 2019. DrugCentral lists 2 molecular targets, including GDF11 and MSTN. Recorded indications include beta Thalassemia and Myelodysplastic syndrome.

• Drug class: Erythroid Maturation Agent.
• Molecular targets: GDF11 and MSTN (2 listed in DrugCentral).
• Indications: beta Thalassemia and Myelodysplastic syndrome.
• ATC code: B03XA06 (Other antianemic preparations).

Indications & contraindications

Drug–disease relationships curated by DrugCentral (OMOP / SNOMED concepts).

Indications (2)

• beta Thalassemia
• Myelodysplastic syndrome

Mechanism & targets

• Established pharmacologic class: Erythroid Maturation Agent

Molecular targets (2)

Target	Gene	Action	Activity
Growth/differentiation factor 11	GDF11	other	Kd 10.13
Growth/differentiation factor 8	MSTN	other	Kd 9.69

Regulatory approvals (2)

Date	Agency	Marketed as	Applicant	Orphan
2020-06-25	EMA	—	Celgene Europe B.V.	Yes
2019-11-08	FDA	—	CELGENE CORP	Yes

ATC classification

ATC code	Therapeutic hierarchy
B03XA06	BLOOD AND BLOOD FORMING ORGANS › ANTIANEMIC PREPARATIONS › OTHER ANTIANEMIC PREPARATIONS › Other antianemic preparations

Pharmacogenomics

Drug-label and guideline annotations from PharmGKB and CPIC.

• FDA label: HBB

Reported adverse-event signals

Strongest FDA FAERS disproportionality signals (log-likelihood ratio). A signal is a statistical association in spontaneous reports, not proof that the drug causes the event.

MedDRA term	LLR
Product storage error	283.6
Death	235.7
Haemoglobin decreased	182.6
Extramedullary haemopoiesis	168.3
Transformation to acute myeloid leukaemia	145.7
Product preparation error	119.5
Product preparation issue	76.9
Haemoglobin increased	59.6
Myelodysplastic syndrome	56.3
Bone pain	50.6
Adverse event	43.1
Full blood count decreased	43.1

Chemical structure

• Molecular formula: —
• Molecular weight: —
• CAS number: 1373715-00-4
• InChIKey: —
• PubChem CID: —

Frequently asked questions

Answers are generated from the structured data on this page, not written by hand.

What is Luspatercept used for?

DrugCentral lists the following indications for Luspatercept: beta Thalassemia and Myelodysplastic syndrome.

How does Luspatercept work?

Its molecular targets in DrugCentral include GDF11 and MSTN.

When was Luspatercept first approved?

Luspatercept was first approved by the FDA in 2019.

What drug class does Luspatercept belong to?

Luspatercept is in the Erythroid Maturation Agent class.

Sources

• DrugCentral drug card 5364 — structure, indications, targets, approvals, pharmacologic classes (CC BY-SA 4.0)
• ChEMBL CHEMBL3039545 — compound record and bioactivity
• DrugBank DB12281 — drug record
• FDA UNII AQK7UBA1LS — substance identity
• DailyMed: FDA labels for Luspatercept — current prescribing information (label search)
• UniProt O95390 (GDF11) — target protein
• UniProt O14793 (MSTN) — target protein
• FDA Adverse Event Reporting System (openFDA FAERS) — adverse-event signals, as aggregated by DrugCentral
• PharmGKB / CPIC — pharmacogenomic label annotations

How this page is built: methodology · editorial policy & corrections.