Details of the Drug

General Information of Drug (ID: DM4GXW3)

Drug Name
Ataluren
Synonyms
Ataluren; 775304-57-9; PTC124; 3-(5-(2-fluorophenyl)-1,2,4-oxadiazol-3-yl)benzoic acid; PTC-124; 3-[5-(2-Fluorophenyl)-1,2,4-oxadiazol-3-yl]benzoic acid; PTC124 (Ataluren); PTC 124; UNII-K16AME9I3V; Ataluren (PTC124); K16AME9I3V; NCGC00168759-02; Translarna; AK-31904; Benzoic acid, 3-[5-(2-fluorophenyl)-1,2,4-oxadiazol-3-yl]-; DSSTox_CID_26776; DSSTox_RID_81895; DSSTox_GSID_46776; Benzoic acid, 3-(5-(2-fluorophenyl)-1,2,4-oxadiazol-3-yl)-; CAS-775304-57-9; Ataluren [USAN:INN]; Translarna (TN); PTC124,Ataluren; Ataluren(ptc124)
Indication
Disease Entry ICD 11 Status REF
Cystic fibrosis CA25 Phase 3 [1]
Dravet syndrome 8A61.11 Phase 2 [2]
Muscular dystrophy 8C70 Phase 2 [2]
Aniridia LA11.3 Phase 1 [1]
Mucopolysaccharidosis 5C56.3 Phase 1 [1]
Duchenne dystrophy 8C70 Application submitted [2]
⏷ Show the Full List of Indication(s)
Affected Organisms
Humans and other mammals
ATC Code
M09AX03: Ataluren
M09AX: Other drugs for disorders of the musculo-skeletal system
M09A: OTHER DRUGS FOR DISORDERS OF THE MUSCULO-SKELETAL SYSTEM
M09: OTHER DRUGS FOR DISORDERS OF THE MUSCULO-SKELETAL SYSTEM
M: MUSCULO-SKELETAL SYSTEM
Drug Type
Small molecular drug
Structure
3D MOL 2D MOL
#Ro5 Violations (Lipinski): 0 Molecular Weight (mw) 284.24
Logarithm of the Partition Coefficient (xlogp) 3.1
Rotatable Bond Count (rotbonds) 3
Hydrogen Bond Donor Count (hbonddonor) 1
Hydrogen Bond Acceptor Count (hbondacc) 6
ADMET Property
Elimination
In the urine, unchanged ataluren and the acyl glucuronide metabolite account for less than 1% and 49%, respectively, of the administered dose [3]
Half-life
The concentration or amount of drug in body reduced by one-half in 2 - 6 hours [3]
Metabolism
The drug is metabolized via the conjugation via uridine diphosphate glucuronosyltransferase (UGT) enzymes (predominantly UGT1A9) in liver and intestine [3]
Chemical Identifiers
Formula
C15H9FN2O3
IUPAC Name
3-[5-(2-fluorophenyl)-1,2,4-oxadiazol-3-yl]benzoic acid
Canonical SMILES
C1=CC=C(C(=C1)C2=NC(=NO2)C3=CC(=CC=C3)C(=O)O)F
InChI
InChI=1S/C15H9FN2O3/c16-12-7-2-1-6-11(12)14-17-13(18-21-14)9-4-3-5-10(8-9)15(19)20/h1-8H,(H,19,20)
InChIKey
OOUGLTULBSNHNF-UHFFFAOYSA-N
Cross-matching ID
PubChem CID
11219835
ChEBI ID
CHEMBL94805
CAS Number
775304-57-9
UNII
K16AME9I3V
DrugBank ID
DB05016
TTD ID
D09IRQ
INTEDE ID
DR0149
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Molecular Interaction Atlas of This Drug


Drug Therapeutic Target (DTT)
DTT Name DTT ID UniProt ID MOA REF
Dystrophin nonsense mutant (DMD) TT2TNRM DMD_HUMAN Modulator [4]

Drug-Metabolizing Enzyme (DME)
DME Name DME ID UniProt ID MOA REF
UDP-glucuronosyltransferase 1A9 (UGT1A9)
Main DME 		DE85D2P UD19_HUMAN Substrate [5]
Molecular Interaction Atlas (MIA) Jump to Detail Molecular Interaction Atlas of This Drug

References

1 Clinical pipeline report, company report or official report of the Pharmaceutical Research and Manufacturers of America (PhRMA)
2 Clinical pipeline report, company report or official report of the Pharmaceutical Research and Manufacturers of America (PhRMA)
3 EMA Product Label
4 URL: http://www.guidetopharmacology.org Nucleic Acids Res. 2015 Oct 12. pii: gkv1037. The IUPHAR/BPS Guide to PHARMACOLOGY in 2016: towards curated quantitative interactions between 1300 protein targets and 6000 ligands. (Ligand id: 7341).
5 Ataluren pharmacokinetics in healthy Japanese and Caucasian subjects. Clin Pharmacol Drug Dev. 2019 Feb;8(2):172-178.