Investigational Drug Details
Drug ID: | D484 |
Drug Name: | Idebenone |
Synonyms: | -- |
Type: | Chemical drug |
DrugBank ID: | DB09081 |
DrugBank Description: | Idebenone is an analogue of ubiquinone used for the treatment of visual impairment in adolescent and adult patients with Leber’s Hereditary Optic Neuropathy (LHON). |
PubChem ID: | -- |
CasNo: | 58186-27-9 |
Repositioning for NAFLD: | Yes |
SMILES: | COC1=C(OC)C(=O)C(CCCCCCCCCCCCO)=C(C)C1=O |
Structure: |
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InChiKey: | VMHWZDULLBLUMS-UHFFFAOYSA-N |
Molecular Weight: | 366.498 |
DrugBank Targets: | -- |
DrugBank MoA: | Idebenone is a synthetic analogue of ubiquinone (also known as Coenzyme Q10), a vital cell antioxidant and essential component of the Electron Transport Chain (ETC). It has been proposed that by interacting with the ETC, idebenone increases ATP production required for mitochondrial function, reduces free radicals, inhibits lipid peroxidation, and consequently protects the lipid membrane and mitochondria from oxidative damage. More specifically, idebenone is thought to transfer electrons directly to complex III of the mitochondrial ETC, thereby circumventing complex I and restoring cellular energy (ATP) generation. |
DrugBank Pharmacology: | -- |
DrugBank Indication: | Idebenone is indicated for use by the European Medicines Agency (EMA) for the treatment of visual impairment in adolescent and adult patients with Leber’s Hereditary Optic Neuropathy (LHON). It is not currently approved for use by either the Food and Drug Administration (USA) or Health Canada. |
Targets: | -- |
Therapeutic Category: | -- |
Clinical Trial Progress: | Phase 1&2 on-going (NCT04669158) |
Latest Progress: | Under clinical trials |

Trial ID | Source ID | Phases | Status | Study Results | Start Date | Last Update Posted | |
---|---|---|---|---|---|---|---|
L0189 | NCT04669158 | Phase 1/Phase 2 | Recruiting | No Results Available | July 30, 2021 | July 23, 2021 | Details |
Target ID | Target Name | GENE | Action | Class | UniProtKB ID | Entry Name |
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Strategy ID | Strategy | Synonyms | Related Targets | Related Drugs |
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Article ID | PMID | Source | Title | |
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A02445 | 34369032 | J Biochem Mol Toxicol | Shc inhibitor idebenone ameliorates liver injury and fibrosis in dietary NASH in mice. | Details |
A13037 | 30171034 | Dis Model Mech | Idebenone and coenzyme Q10 are novel PPARα/γ ligands, with potential for treatment of fatty liver diseases. | Details |