ataluren 🐶 Veterinary Use | Indications/Contra | FAERs-F | FAERs-M | Orange Bk | BioActivity |

Stem definitionDrug idCAS RN
4872 775304-57-9

Description:

MoleculeDescription

Molfile Inchi Smiles

Synonyms:

  • ataluren
  • translarna
  • PTC124
A nonsense mutation in DNA results in a premature stop codon within an mRNA. This premature stop codon in the mRNA causes disease by terminating translation before a full-length protein is generated. Ataluren enables ribosomal readthrough of mRNA containing such a premature stop codon, resulting in production of a full-length protein.
  • Molecular weight: 284.25
  • Formula: C15H9FN2O3
  • CLOGP: 3.67
  • LIPINSKI: 0
  • HAC: 5
  • HDO: 1
  • TPSA: 76.22
  • ALOGS: -3.38
  • ROTB: 3

Drug dosage:

DoseUnitRoute
2.80 g O

ADMET properties:

None

Approvals:

DateAgencyCompanyOrphan
July 31, 2014 EMA PTC Therapeutics International Limited

FDA Adverse Event Reporting System (Female)

None

FDA Adverse Event Reporting System (Male)

None

FDA Adverse Event Reporting System (Geriatric)

None

FDA Adverse Event Reporting System (Pediatric)

None

Pharmacologic Action:

SourceCodeDescription
ATC M09AX03 MUSCULO-SKELETAL SYSTEM
OTHER DRUGS FOR DISORDERS OF THE MUSCULO-SKELETAL SYSTEM
OTHER DRUGS FOR DISORDERS OF THE MUSCULO-SKELETAL SYSTEM
Other drugs for disorders of the musculo-skeletal system

Drug Use | Suggest Off label Use Form| |View source of the data|

DiseaseRelationSNOMED_IDDOID
Duchenne muscular dystrophy indication 76670001 DOID:11723




🐶 Veterinary Drug Use

None

🐶 Veterinary products

None

Acid dissociation constants calculated using MoKa v3.0.0

Dissociation levelDissociation constantType (acidic/basic)
pKa1 3.43 acidic

Orange Book patent data (new drug applications)

None

Orange Book exclusivity data (new drug applications)

None

Bioactivity Summary:

TargetClassPharosUniProtActionTypeActivity value
(-log[M])
Mechanism
action
Bioact sourceMoA source
Flavin reductase (NADPH) Enzyme Kd 6.70 CHEMBL

External reference:

IDSource
K16AME9I3V UNII
D09323 KEGG_DRUG
C2930764 UMLSCUI
CHEBI:94805 CHEBI
JBF PDB_CHEM_ID
CHEMBL256997 ChEMBL_ID
11219835 PUBCHEM_CID
DB05016 DRUGBANK_ID
8956 INN_ID
C515878 MESH_SUPPLEMENTAL_RECORD_UI
7341 IUPHAR_LIGAND_ID
015935 NDDF
1263915001 SNOMEDCT_US
718843009 SNOMEDCT_US

Pharmaceutical products:

None