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Agalsidase Alfa

CHEMBL2108214 Phase 4 승인됨 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.

분자량

959.2000 g/mol

TPSA

257.00 Ų

작용 기전

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

Pharmacokinetics (PK)

Pharmacodynamics (PD)

기전

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

2D 구조

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

                    

                    
                    

                        
회전 가능 결합

                        
25

                    

                    
                    
                    

                        
무거운 원자

                        
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.

    



                
            


            
            
            

                
자주 묻는 질문

                

                    
                    

                        
                        

                            
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

    
    
        
        
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                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.




        


        
        
    


    



    
의학적 면책조항

    

        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.