Molecule Details
InChIKeyOOUGLTULBSNHNF-UHFFFAOYSA-N
Canonical SMILESO=C(O)c1cccc(-c2noc(-c3ccccc3F)n2)c1
Clinical Status Data-mined Candidate
Targets (Human+Pathogen)2
Pfam Stratification Cross-Family
Avg pChEMBL6.61
SourceChEMBL;BindingDB
2D Structure
2D structure
Activity Profile
DrugBank Annotations
DrugBank ID DB05016
Drug NameAtaluren
CAS Number775304-57-9
Groups approved withdrawn
ATC Codes M09AX03
DescriptionAtaluren is a novel, orally administered drug that targets nonsense mutations. Ataluren is approved for use by the European Medicines Agency to treat Duchenne Muscular Dystrophy in patients aged 5 years and older who are able to walk. More specifically, ataluren is used in the small group of patient...

Categories: Musculo-Skeletal System OAT1/SLC22A6 inhibitors OAT3/SLC22A8 Inhibitors OATP1B3 inhibitors Oxazoles UGT1A9 Substrates
Cross-references: ChEBI: 94805 CHEMBL256997 ChemSpider: 9394889 D09323 PDB: JBF PharmGKB: PA166151864 PubChem:11219835 PubChem:175426931 Wikipedia: Ataluren ZINC: ZINC000013831791
Target Activities (2)
Target Gene Organism Category Pfam pChEMBL Type Source
P30043 BLVRB Homo sapiens Human PF13460 6.7 Kd ChEMBL;BindingDB
Q9UBN7 HDAC6 Homo sapiens Human PF00850 PF02148 6.5 IC50 ChEMBL
DrugBank Target Actions (5)
Target Gene Target Name Action Type
Q4U2R8 SLC22A6 Solute carrier family 22 member 6 inhibitor enzymes
Q8TCC7 SLC22A8 Organic anion transporter 3 inhibitor enzymes
Q9NPD5 SLCO1B3 Solute carrier organic anion transporter family member 1B3 inhibitor enzymes
O60656 O60656 UDP-glucuronosyltransferase 1A9 substrate enzymes
P11532 P11532 Dystrophin modulator targets