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Trofinetide

CHEMBL197084 Phase 4 Approuvé Small molecule
Half-Life
—
Bioavailability
—
Protein Binding
—
Molecular Weight
315.3 g/mol
LogP
-3.9
Phase
4

Trofinetide is a synthetic analog of glycine-proline-glutamate (GPE), a naturally occurring N-terminal tripeptide fragment of insulin-like growth factor-1, that modulates neuroinflammation and synaptic function in the brain. It is approved for the treatment of Rett syndrome in adults and pediatric patients, a rare neurodevelopmental disorder caused by MECP2 gene mutations, where it reduces core symptoms. Its mechanism involves reducing neuroinflammation and supporting synaptic integrity in affected neural circuits.

Masse moléculaire

315,3200 g/mol

LogP

-3,90

TPSA

150,00 Ų

Règle des 5 de Lipinski

Conforme

Pharmacokinetics (PK)

Pharmacodynamics (PD)

Structure 2D

SVG PNG

Cite this structure


                        

                            
                            
                        

                    

                


                
                

                    

                    

                        
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SMILES

                    
C[C@@]1(C(=O)N[C@@H](CCC(=O)O)C(=O)O)CCCN1C(=O)CN

                

                

                
                

                    
InChI

                    
InChI=1S/C13H21N3O6/c1-13(5-2-6-16(13)9(17)7-14)12(22)15-8(11(20)21)3-4-10(18)19/h8H,2-7,14H2,1H3,(H,15,22)(H,18,19)(H,20,21)/t8-,13-/m0/s1

                

                

                

                    
                    

                        
Molecular Formula

                        
C13H21N3O6

                    

                    
                    

                        
HBD / HBA

                        
4 / 7

                    

                    
                    

                        
Liaisons Rotatives

                        
7

                    

                    
                    
                    

                        
Atomes Lourds

                        
22

                    

                    
                

            


            
            

                
                


    
    
        
    
    
    
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

                

                    
                    

                        
                        

                            
Trofinetide is a synthetic analog of glycine-proline-glutamate (GPE), a naturally occurring N-terminal tripeptide fragment of insulin-like growth factor-1, that modulates neuroinflammation and synaptic function in the brain. It is approved for the treatment of Rett syndrome in adults and pediatric patients, a rare neurodevelopmental disorder caused by MECP2 gene mutations, where it reduces core symptoms. Its mechanism involves reducing neuroinflammation and supporting synaptic integrity in affected neural circuits.

                        

                    

                    

                    

                    

                    

                    

                        
                        

                            
Yes, Trofinetide 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).
                CHEMBL197084.
                Open-access bioactivity database.
            
        
    • 
        
        
        
  • 
            
            
                PubChem — National Center for Biotechnology Information (NCBI).
                CID 11318905.
                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.