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Tafamidis

CHEMBL2103837 Phase 4 已批准 Small molecule
Half-Life
—
Bioavailability
—
Protein Binding
—
Molecular Weight
308.1 g/mol
LogP
4.2
Phase
4

Tafamidis is a selective transthyretin (TTR) stabilizer that binds to the thyroxine-binding sites of TTR tetramers, preventing their dissociation into monomers and halting the amyloidogenic cascade responsible for transthyretin amyloid cardiomyopathy (ATTR-CM). By kinetically stabilizing the native TTR tetramer, it reduces misfolding and fibril deposition in cardiac tissue, slowing progression of cardiomyopathy. It is the first disease-modifying therapy approved for ATTR-CM and demonstrates mortality reduction in clinical trials.

分子量

308.1000 g/mol

LogP

4.20

TPSA

63.30 Ų

Lipinski 五规则

符合

作用机制

Stabilizes the transthyretin (TTR) protein tetramer, preventing its dissociation into monomers that misfold and aggregate into amyloid fibrils in cardiac tissue.

Pharmacokinetics (PK)

Pharmacodynamics (PD)

机制

Stabilizes the transthyretin (TTR) protein tetramer, preventing its dissociation into monomers that misfold and aggregate into amyloid fibrils in cardiac tissue.

二维结构

SVG PNG

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SMILES

                    
O=C(O)c1ccc2nc(-c3cc(Cl)cc(Cl)c3)oc2c1

                

                

                
                

                    
InChI

                    
InChI=1S/C14H7Cl2NO3/c15-9-3-8(4-10(16)6-9)13-17-11-2-1-7(14(18)19)5-12(11)20-13/h1-6H,(H,18,19)

                

                

                

                    
                    

                        
Molecular Formula

                        
C14H7Cl2NO3

                    

                    
                    

                        
HBD / HBA

                        
1 / 4

                    

                    
                    

                        
可旋转键数

                        
2

                    

                    
                    
                    

                        
重原子数

                        
20

                    

                    
                

            


            
            

                
                


    
    
        
    
    
    
No targets recorded

    
    
Target interaction data is not yet available for this drug.

    



                
            


            
            

                
                


    
    
        
    
    
    
No interactions recorded

    
    
Drug interaction data is not yet available for this compound.

    



                
            


            
            

                
                


    
    
        
    
    
    
No side effects recorded

    
    
Side effect data is not yet available for this drug.

    



                
            


            
            
            

                
常见问题

                

                    
                    

                        
                        

                            
Tafamidis is a selective transthyretin (TTR) stabilizer that binds to the thyroxine-binding sites of TTR tetramers, preventing their dissociation into monomers and halting the amyloidogenic cascade responsible for transthyretin amyloid cardiomyopathy (ATTR-CM). By kinetically stabilizing the native TTR tetramer, it reduces misfolding and fibril deposition in cardiac tissue, slowing progression of cardiomyopathy. It is the first disease-modifying therapy approved for ATTR-CM and demonstrates mortality reduction in clinical trials.

                        

                    

                    

                    

                    
                    

                        
                        

                            
Stabilizes the transthyretin (TTR) protein tetramer, preventing its dissociation into monomers that misfold and aggregate into amyloid fibrils in cardiac tissue.

                        

                    

                    

                    

                    

                        
                        

                            
Yes, Tafamidis is an approved drug. It has reached clinical phase 4. It is classified as a Small molecule.

                        

                    

                

            

            

            
            

            
            

            
            
{# References & Data Sources section for drug detail pages.
   Renders standard pharmacological database links plus the drug's data_sources field. #}


    
References & Data Sources

    
    
        
        
  • 
            
            
                ChEMBL — European Bioinformatics Institute (EBI).
                CHEMBL2103837.
                Open-access bioactivity database.
            
        
    • 
        
        
        
  • 
            
            
                PubChem — National Center for Biotechnology Information (NCBI).
                CID 11001318.
                Chemical information database.
            
        
    • 
        
        
        
        
    

    
Data aggregated from publicly available pharmacological databases. Last updated 2026-03-28.




        


        
        
    


    



    
医疗免责声明

    

        This content is for educational and informational purposes only. It is not a substitute for professional medical advice, diagnosis, or treatment. Always consult a qualified healthcare provider before making medication decisions.
    

    

        Data sources:
        ChEMBL,
        PubChem,
        DailyMed.