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Omaveloxolone

CHEMBL4303525 Phase 4 Zugelassen Small molecule
Half-Life
—
Bioavailability
—
Protein Binding
—
Molecular Weight
554.7 g/mol
LogP
6.7
Phase
4

Friedreich's ataxia had no disease-directed therapy before omaveloxolone, which is the first approved agent aimed at the underlying dysfunction of that rare progressive neurological disorder. The drug activates a cytoprotective transcriptional pathway that counters oxidative stress and inflammation, processes implicated in the mitochondrial impairment characteristic of the disease. Protecting neurones from that oxidative burden addresses a mechanism upstream of the neurodegeneration rather than the symptoms alone, which is what distinguishes it from supportive treatment. The compound is a small molecule with the formula C33H44F2N2O3 and a molecular weight close to 554.7 g/mol. It is approved and has completed the final phase of clinical development.

Used to treat Friedreich's ataxia, a rare progressive neurological disease, this medication activates a pathway that helps protect nerve cells from oxidative stress and inflammation. It is the first approved treatment specifically targeting the underlying dysfunction in this condition.

Molekularmasse

554,7000 g/mol

LogP

6,70

TPSA

87,00 Ų

Lipinski-Regel der Fünf

Nicht bestanden

Therapeutische Bereiche

Neurology

Pharmacokinetics (PK)

Pharmacodynamics (PD)

2D-Struktur

SVG PNG

Cite this structure


                        

                            
                            
                        

                    

                


                
                

                    

                    

                        
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SMILES

                    
CC1(C)CC[C@]2(NC(=O)C(C)(F)F)CC[C@]3(C)[C@H](C(=O)C=C4[C@@]5(C)C=C(C#N)C(=O)C(C)(C)[C@@H]5CC[C@]43C)[C@@H]2C1

                

                

                
                

                    
InChI

                    
InChI=1S/C33H44F2N2O3/c1-27(2)11-13-33(37-26(40)32(8,34)35)14-12-31(7)24(20(33)17-27)21(38)15-23-29(5)16-19(18-36)25(39)28(3,4)22(29)9-10-30(23,31)6/h15-16,20,22,24H,9-14,17H2,1-8H3,(H,37,40)/t20-,22-,24-,29-,30+,31+,33-/m0/s1

                

                

                

                    
                    

                        
Molecular Formula

                        
C33H44F2N2O3

                    

                    
                    

                        
HBD / HBA

                        
1 / 6

                    

                    
                    

                        
Rotierbare Bindungen

                        
2

                    

                    
                    
                    

                        
Schwere Atome

                        
40

                    

                    
                

            


            
            

                
                


    
    
        
    
    
    
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

                

                    
                    

                        
                        

                            
Used to treat Friedreich's ataxia, a rare progressive neurological disease, this medication activates a pathway that helps protect nerve cells from oxidative stress and inflammation. It is the first approved treatment specifically targeting the underlying dysfunction in this condition.

                        

                    

                    

                    

                    

                    

                    

                        
                        

                            
Yes, Omaveloxolone 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).
                CHEMBL4303525.
                Open-access bioactivity database.
            
        
    • 
        
        
        
  • 
            
            
                PubChem — National Center for Biotechnology Information (NCBI).
                CID 71811910.
                Chemical information database.
            
        
    • 
        
        
        
        
    

    
Data aggregated from publicly available pharmacological databases. Last updated 2026-08-20.




        


        
        
    


    



    
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.