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Agalsidase Alfa

CHEMBL2108214 Phase 4 Zugelassen Enzyme
Half-Life
—
Bioavailability
—
Protein Binding
—
Molecular Weight
959.2 g/mol
LogP
—
Phase
4

A synthetic enzyme replacement therapy designed to substitute for alpha-galactosidase A, an enzyme that is deficient in people with Fabry disease, a rare inherited metabolic disorder. It helps break down a fatty substance called globotriaosylceramide that otherwise accumulates in blood vessel walls and organs, causing progressive damage.

Molekularmasse

959,2000 g/mol

TPSA

257,00 Ų

Wirkmechanismus

Provides exogenous replacement of the deficient enzyme, restoring the metabolic pathway and clearing accumulated substrates from affected tissues.

Pharmacokinetics (PK)

Pharmacodynamics (PD)

Mechanismus

Provides exogenous replacement of the deficient enzyme, restoring the metabolic pathway and clearing accumulated substrates from affected tissues.

2D-Struktur

SVG PNG

Cite this structure


                        

                            
                            
                        

                    

                


                
                

                    

                    

                        
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SMILES

                    
CCCCCCCC(=O)N[C@H](CN1CCCC1)[C@@H](C2=CC3=C(C=C2)OCCO3)O.CCCCCCCC(=O)N[C@H](CN1CCCC1)[C@@H](C2=CC3=C(C=C2)OCCO3)O.[C@@H]([C@H](C(=O)O)O)(C(=O)O)O

                

                

                
                

                    
InChI

                    
InChI=1S/2C23H36N2O4.C4H6O6/c2*1-2-3-4-5-6-9-22(26)24-19(17-25-12-7-8-13-25)23(27)18-10-11-20-21(16-18)29-15-14-28-20;5-1(3(7)8)2(6)4(9)10/h2*10-11,16,19,23,27H,2-9,12-15,17H2,1H3,(H,24,26);1-2,5-6H,(H,7,8)(H,9,10)/t2*19-,23-;1-,2-/m111/s1

                

                

                

                    
                    

                        
Molecular Formula

                        
C50H78N4O14

                    

                    
                    

                        
HBD / HBA

                        
8 / 16

                    

                    
                    

                        
Rotierbare Bindungen

                        
25

                    

                    
                    
                    

                        
Schwere Atome

                        
68

                    

                    
                

            


            
            

                
                


    
    
        
    
    
    
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.

    



                
            


            
            
            

                
Häufig gestellte Fragen

                

                    
                    

                        
                        

                            
A synthetic enzyme replacement therapy designed to substitute for alpha-galactosidase A, an enzyme that is deficient in people with Fabry disease, a rare inherited metabolic disorder. It helps break down a fatty substance called globotriaosylceramide that otherwise accumulates in blood vessel walls and organs, causing progressive damage.

                        

                    

                    

                    

                    
                    

                        
                        

                            
Provides exogenous replacement of the deficient enzyme, restoring the metabolic pathway and clearing accumulated substrates from affected tissues.

                        

                    

                    

                    

                    

                        
                        

                            
Yes, Agalsidase Alfa is an approved drug. It has reached clinical phase 4. It is classified as a Enzyme.

                        

                    

                

            

            

            
            

            
            

            
            
{# 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).
                CHEMBL2108214.
                Open-access bioactivity database.
            
        
    • 
        
        
        
  • 
            
            
                PubChem — National Center for Biotechnology Information (NCBI).
                CID 52918379.
                Chemical information database.
            
        
    • 
        
        
        
        
    

    
Data aggregated from publicly available pharmacological databases. Last updated 2026-03-04.




        


        
        
    


    



    
Medizinischer Haftungsausschluss

    

        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.