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Luspatercept | CAS No: 1373715-00-4 | GMP-certified suppliers
A medication that treats anemia in adults with beta thalassemia or myelodysplastic syndromes requiring regular red blood cell transfusions and supports erythroid maturation.
Therapeutic categories
Activin ReceptorsAmino Acids, Peptides, and ProteinsAntianemia DrugsAntianemic PreparationsBlood and Blood Forming OrgansEnzymes
Generic name
Luspatercept
Molecule type
biotech
CAS number
1373715-00-4
DrugBank ID
DB12281
Approval status
Approved drug, Investigational drug
ATC code
B03XA06
Primary indications
- Luspatercept is indicated for the treatment of:
- Anemia in adults with beta thalassemia who require regular red blood cell transfusions
- Anemia without previous erythropoiesis stimulating agent use (ESA-naïve) in adult patients with very low- to intermediate-risk myelodysplastic syndromes (MDS) who may require regular red blood cell (RBC) transfusions
Product Snapshot
- Luspatercept is a subcutaneous injectable peptide formulation supplied as a lyophilized powder for solution
- It is primarily used for treating anemia associated with beta thalassemia and specific myelodysplastic syndromes requiring red blood cell transfusions
- The product holds regulatory approvals in the US, EU, and Canada, with both approved and investigational indications
Clinical Overview
Luspatercept (CAS Number 1373715-00-4) is a recombinant fusion protein consisting of a modified extracellular domain of activin receptor type IIB linked to the Fc domain of human IgG1. It is approved for the treatment of anemia associated with beta thalassemia in adult patients who require regular red blood cell transfusions. Additionally, luspatercept is indicated for anemia in adult patients with very low- to intermediate-risk myelodysplastic syndromes (MDS) who are either erythropoiesis-stimulating agent (ESA) naïve or have failed prior ESA therapy, particularly those with ring sideroblasts or myelodysplastic/myeloproliferative neoplasm with ring sideroblasts and thrombocytosis.
The pharmacological action of luspatercept targets late-stage erythropoiesis, distinguishing it from traditional ESAs such as darbepoetin alfa and epoetin alfa, which stimulate early erythroid progenitors. Luspatercept functions as a ligand trap by binding to multiple ligands within the transforming growth factor beta (TGF-β) superfamily, which normally act as negative regulators of erythroid maturation. By inhibiting these ligands, luspatercept alleviates ineffective erythropoiesis characteristic of beta thalassemia and relevant myelodysplastic conditions. The precise ligands targeted are not fully defined; however, experimental data exclude growth differentiation factor 11 (GDF11) as a target.
Pharmacokinetic and metabolism data indicate that luspatercept is administered by subcutaneous injection with typical protein therapeutic properties. Detailed ADME parameters are not publicly disclosed but are consistent with large recombinant proteins, involving proteolytic catabolism rather than classical small molecule metabolism.
Safety considerations include reports of thromboembolic events such as deep vein thrombosis and pulmonary embolism in patients with beta thalassemia. Risk mitigation may involve thromboprophylaxis for patients with elevated baseline risk. Embryo-fetal toxicity has been observed in animal studies, necessitating contraception in women of childbearing potential during and for at least three months post-therapy. The development of extramedullary hematopoietic masses also warrants clinical monitoring.
Luspatercept is marketed primarily under the brand name Reblozyl®. Given its recombinant protein nature, sourcing high-quality active pharmaceutical ingredient requires adherence to stringent biotechnological production standards, including robust cell line characterization, purification processes, and thorough impurity profiling. Suppliers should comply with current Good Manufacturing Practices (cGMP) and provide comprehensive documentation to support regulatory submissions and ensure product consistency.
The pharmacological action of luspatercept targets late-stage erythropoiesis, distinguishing it from traditional ESAs such as darbepoetin alfa and epoetin alfa, which stimulate early erythroid progenitors. Luspatercept functions as a ligand trap by binding to multiple ligands within the transforming growth factor beta (TGF-β) superfamily, which normally act as negative regulators of erythroid maturation. By inhibiting these ligands, luspatercept alleviates ineffective erythropoiesis characteristic of beta thalassemia and relevant myelodysplastic conditions. The precise ligands targeted are not fully defined; however, experimental data exclude growth differentiation factor 11 (GDF11) as a target.
Pharmacokinetic and metabolism data indicate that luspatercept is administered by subcutaneous injection with typical protein therapeutic properties. Detailed ADME parameters are not publicly disclosed but are consistent with large recombinant proteins, involving proteolytic catabolism rather than classical small molecule metabolism.
Safety considerations include reports of thromboembolic events such as deep vein thrombosis and pulmonary embolism in patients with beta thalassemia. Risk mitigation may involve thromboprophylaxis for patients with elevated baseline risk. Embryo-fetal toxicity has been observed in animal studies, necessitating contraception in women of childbearing potential during and for at least three months post-therapy. The development of extramedullary hematopoietic masses also warrants clinical monitoring.
Luspatercept is marketed primarily under the brand name Reblozyl®. Given its recombinant protein nature, sourcing high-quality active pharmaceutical ingredient requires adherence to stringent biotechnological production standards, including robust cell line characterization, purification processes, and thorough impurity profiling. Suppliers should comply with current Good Manufacturing Practices (cGMP) and provide comprehensive documentation to support regulatory submissions and ensure product consistency.
Identification & chemistry
| Generic name | Luspatercept |
|---|---|
| Molecule type | Biotech |
| CAS | 1373715-00-4 |
| UNII | AQK7UBA1LS |
| DrugBank ID | DB12281 |
Pharmacology
| Summary | Luspatercept is a fusion protein that targets the transforming growth factor beta (TGF-β) superfamily ligands involved in negative regulation of late-stage erythropoiesis. By acting as a ligand trap for these signaling molecules, it reduces their inhibition of erythroid maturation, thereby improving ineffective erythropoiesis in conditions such as beta thalassemia and certain myelodysplastic syndromes. Its pharmacodynamic effects center on enhancing red blood cell production through modulation of intracellular Smad2/3 signaling pathways. |
|---|---|
| Mechanism of action | Beta thalassemia is a genetic red blood cell disorder caused by mutations in the β-globin gene - these mutations cause oxidative stress and premature apoptosis of erythroblasts, thereby leading to ineffective erythropoesis. The transforming growth factor beta (TGF-β) superfamily of endogenous ligands (including activins, growth differentiation factors, and bone morphogenetic proteins) are involved in the inhibition of erythroid differentiation via activation of the Smad2/3 subfamily of intracellular effectors. Luspatercept is a fusion protein comprising a modified extracellular domain of activin receptor type IIB (a target for many TGF-β ligands) fused to the FC domain of human IgG1. Luspatercept ameliorates ineffective erythropoiesis in patients with beta thalassemia by acting as a "ligand trap" for various members of the TGF-β superfamily, preventing their downstream signalling and subsequent inhibition of late-stage erythroid maturation. The specific members of the TGF-β superfamily targeted by luspatercept are currently unknown, though growth differentiation factor 11 (GDF11) has been experimentally excluded as a potential target. |
| Pharmacodynamics | Luspatercept binds to, and inhibits, several ligands that act as negative regulators of late-stage erythropoiesis, thereby alleviating the ineffective erythropoiesis observed in patients with beta thalassemia. Thromboembolic events (e.g. deep vein thrombosis, pulmonary embolism, ischemic stroke) have been reported in patients with beta thalassemia receiving luspatercept - patients with a greater baseline risk of thromboembolism may benefit from concomitant thromboprophylaxis while undergoing therapy with luspatercept. Luspatercept may carry some degree of embryo-fetal toxicity and should therefore be avoided in pregnancy. Women of child-bearing age should use an effective form of contraception during therapy and for 3 months after completion of therapy. Luspatercept may also lead to the development of extramedullary hematopoietic (EMH) masses in adult patients with transfusion-dependent beta-thalassemia. |
ADME / PK
| Absorption | At doses of 1 mg/kg and 1.25 mg/kg, the average steady-state AUC was 126 day•μg/mL and 157 day•μg/mL and the average C<sub>max</sub> was 8.17 μg/mL and 10.2 μg/mL, respectively. Steady-state was reached after 3 doses given every 3 weeks. T<sub>max</sub> is reached approximately 7 days after administration. Absorption pharmacokinetics do not appear to be affected by the site of subcutaneous injection. |
|---|---|
| Half-life | The average half-life of luspatercept is approximately 11 days. |
| Metabolism | As luspatercept is a fusion protein, it is expected to undergo catabolism into amino acids by general protein degradation processes. |
| Volume of distribution | The average apparent volume of distribution is 7.1 L. |
| Clearance | The total apparent clearance of luspatercept is 0.44 L/day. |
Formulation & handling
- Luspatercept is a biotech peptide formulated as a lyophilized powder for subcutaneous injection, requiring reconstitution prior to administration.
- The solid form and peptide nature necessitate careful handling to maintain stability, avoiding exposure to extreme temperatures and moisture.
- Formulation considerations include preservation of protein integrity during manufacturing and storage to ensure efficacy and safety.
Regulatory status
| Lifecycle | The active pharmaceutical ingredient (API) is off-patent in the US, EU, and Canada, with generic versions widely available and established market presence in these regions. Market activities primarily focus on lifecycle management and competitive positioning within mature regulatory environments. |
|---|
| Markets | US, EU, Canada |
|---|
Supply Chain
| Supply chain summary | Luspatercept is primarily manufactured by a limited number of originator companies with branded products present in key markets including the US, EU, and Canada. The consistent presence of the branded product Reblozyl across these regions indicates established global distribution. Patent expiry information is not provided here, so the status of generic competition cannot be assessed. |
|---|
Safety
| Toxicity | Repeat-dose toxicity studies in juvenile rats showed an increase in the development of hematologic malignancies at doses of 10 mg/kg, a dose approximately 8-fold higher than the maximum recommended human dose (MRHD). Fertility studies in rats observed effects on female (but not male) fertility at doses approximately 7-fold higher than the MRHD. |
|---|
High Level Warnings:
- Repeat-dose toxicity in juvenile rats indicated increased hematologic malignancies at doses approximately 8-fold higher than the MRHD
- Fertility assessments revealed effects on female fertility at doses around 7-fold above the MRHD
- No impact observed on male fertility
Luspatercept is a type of Other substances
The pharmaceutical industry encompasses a diverse range of active pharmaceutical ingredients (APIs) that are used in the production of various medications. One category of APIs is known as other substances. This category includes substances that do not fall under the conventional classifications such as antibiotics, analgesics, or antihypertensives.
Other substances in pharmaceutical APIs consist of a broad array of chemical compounds with unique properties and applications. These substances play a crucial role in the formulation and development of specialized medications, catering to specific therapeutic needs. The category encompasses various substances like excipients, solvents, stabilizers, and pH adjusters.
Excipients are inert substances that aid in the manufacturing process and enhance the stability, bioavailability, and patient acceptability of pharmaceutical formulations. Solvents are used to dissolve other ingredients and facilitate their incorporation into the final product. Stabilizers ensure the integrity and shelf life of medications by preventing degradation or chemical changes. pH adjusters help maintain the desired pH level of a formulation, which can influence the drug's efficacy and stability.
Pharmaceutical manufacturers carefully select and incorporate specific other substances into their formulations, adhering to regulatory guidelines and quality standards. These substances undergo rigorous testing and evaluation to ensure their safety, efficacy, and compatibility with the desired pharmaceutical product. By employing other substances in API formulations, pharmaceutical companies can optimize drug delivery, improve patient compliance, and enhance therapeutic outcomes.
In summary, the other substances category of pharmaceutical APIs comprises a diverse range of chemicals, including excipients, solvents, stabilizers, and pH adjusters. These substances contribute to the formulation, stability, and performance of medications, enabling pharmaceutical manufacturers to develop specialized products that meet specific therapeutic requirements.
