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Tafamidis

CHEMBL2103837 Phase 4 Approuvé 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.

Masse moléculaire

308,1000 g/mol

LogP

4,20

TPSA

63,30 Ų

Règle des 5 de Lipinski

Conforme

Mécanisme d'action

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)

Mécanisme

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

Structure 2D

SVG PNG

Cite this structure


                        

                            
                            
                        

                    

                


                
                

                    

                    

                        
Embed this structure

                        

                        

                            
                            
                        

                    

                

                

                
                

                    
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

                    

                    
                    

                        
Liaisons Rotatives

                        
2

                    

                    
                    
                    

                        
Atomes Lourds

                        
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.

    



                
            


            
            
            

                
Foire aux questions

                

                    
                    

                        
                        

                            
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.




        


        
        
    


    



    
Avertissement médical

    

        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.