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Ezutromid
Ezutromid is a small molecule pharmaceutical. It is currently being investigated in clinical studies. It is known to target aryl hydrocarbon receptor.
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Commercial
Therapeutic Areas
No data
Trade Name
FDA
EMA
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Drug Products
FDA
EMA
New Drug Application (NDA)
New Drug Application (NDA)
Abbreviated New Drug Application (ANDA)
Abbreviated New Drug Application (ANDA)
No data
Labels
FDA
EMA
No data
Indications
FDA
EMA
No data
Agency Specific
FDA
EMA
No data
Patent Expiration
No data
ATC Codes
No data
HCPCS
No data
Clinical
Clinical Trials
3 clinical trials
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Indications Phases 4
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Indications Phases 3
No data
Indications Phases 2
Indication
MeSH
Ontology
ICD-10
Ph 1
Ph 2
Ph 3
Ph 4
Other
Total
Duchenne muscular dystrophyD020388213
Indications Phases 1
No data
Indications Without Phase
No data
Epidemiology
Epidemiological information for investigational and approved indications
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Drug
General
Drug common nameEZUTROMID
INNezutromid
Description
Ezutromid is an orally administered small molecule utrophin modulator involved in a Phase 2 clinical trial produced by Summit Therapeutics for the treatment of Duchenne muscular dystrophy (DMD). DMD is a fatal x-linked recessive disease affecting approximately 1 in 5000 males and is a designated orphan disease by the FDA and European Medicines Agency. Approximately 1/3 of the children obtain DMD as a result of spontaneous mutation in the dystrophin gene and have no family history of the disease. Dystrophin is a vital component of mature muscle function, and therefore DMD patients have multifarious forms of defunct or deficient dystrophin proteins that all manifest symptomatically as muscle necrosis and eventually organ failure. Ezutromid is theorized to maintain utrophin, a protein functionally and structurally similar to dystrophin that precedes and is replaced by dystrophin during development. Utrophin and dystrophin are reciprocally expressed, and are found in different locations in a mature muscle cell. However, in dystrophin-deficient patients, utrophin was found to be upregulated and is theorized to replace dystrophin in order to maintain muscle fibers. Ezutromid is projected to have the potential to treat all patients suffering with DMD as it maintains the production of utrophin to counteract the lack of dystrophin to retard muscle degeneration. Both the FDA and European Medicines Agency has given ezutromid an orphan drug designation. The FDA Office of Orphan Products and Development offers an Orphan Drug Designation program (ODD) that allows drugs aimed to treat diseases that affect less than 200,000 people in the U.S. monetary incentives such as a period of market exclusivity, tax incentives, and expedited approval processes.
Classification
Small molecule
Drug class
Image (chem structure or protein)
Structure (InChI/SMILES or Protein Sequence)
CCS(=O)(=O)c1ccc2oc(-c3ccc4ccccc4c3)nc2c1
Identifiers
PDB
CAS-ID945531-77-1
RxCUI
ChEMBL IDCHEMBL1773683
ChEBI ID
PubChem CID25109292
DrugBankDB12888
UNII ID645R07LF0D (ChemIDplus, GSRS)
Target
Agency Approved
No data
Alternate
AHR
AHR
Organism
Homo sapiens
Gene name
AHR
Gene synonyms
BHLHE76
NCBI Gene ID
Protein name
aryl hydrocarbon receptor
Protein synonyms
ah receptor, AH-receptor, aromatic hydrocarbon receptor, bHLHe76, Class E basic helix-loop-helix protein 76
Uniprot ID
Mouse ortholog
Ahr (11622)
aryl hydrocarbon receptor (P30561)
Variants
Clinical Variant
No data
Financial
No data
Trends
PubMed Central
Top Terms for Disease or Syndrome:
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Additional graphs summarizing 46 documents
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Safety
Black-box Warning
No Black-box warning
Adverse Events
Top Adverse Reactions
0 adverse events reported
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