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Accession NumberDB05016
TypeSmall Molecule
DescriptionAtaluren is a novel, orally administered drug that targets nonsense mutations. It is used as a treatment for Duchenne muscular dystrophy (DMD) and cystic fibrosis (CF), and has the potential to treat a number of other genetic disorders caused by nonsense mutations. On 23 May 2014 ataluren received a positive opinion from the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA). Translarna was first available in Germany, the first EU country to launch the new medicine. In August 2014, ataluren received market authorization from the European Commission to treat patients with nonsense mutation Duchenne muscular dystrophy. A confirmatory phase III clinical trial is ongoing. The drug does not yet have approval by the US Food and Drug Administration.
External IDs PTC124
Product Ingredients Not Available
Approved Prescription Products
NameDosageStrengthRouteLabellerMarketing StartMarketing End
TranslarnaGranule125 mgOralPtc Therapeutics2014-07-31Not applicableEu
TranslarnaGranule250 mgOralPtc Therapeutics2014-07-31Not applicableEu
TranslarnaGranule1000 mgOralPtc Therapeutics2014-07-31Not applicableEu
Approved Generic Prescription ProductsNot Available
Approved Over the Counter ProductsNot Available
Unapproved/Other Products Not Available
International Brands
TranslarnaPTC Therapeutics
Brand mixturesNot Available
CAS number775304-57-9
WeightAverage: 284.242
Monoisotopic: 284.059720369
Chemical FormulaC15H9FN2O3
3-[5-(2-fluorophenyl)-1,2,4-oxadiazol-3-yl]benzoic acid
IndicationFor the treatment of cystic fibrosis and muscular dystrophy.
Structured Indications Not Available
PharmacodynamicsNot Available
Mechanism of actionAtaluren allowed the cellular machinery to bypass the nonsense mutation, continue the translation process, and thereby restore the production of a full-length, functional protein. The research on the effects of Ataluren on the translation and stability of nonsense-containing mRNA in vitor show that Ataluren promoted readthrough at each of the nonsense codons, showing maximal activity with UGA, while having no effect on mRNA levels. Unlike the stable cell line assays, Ataluren did not discriminate significantly between the UAG and UAA mRNAs. Ataluren was a more potent nonsense-suppressing agent than gentamicin, and exhibited 4- to 15-fold stimulation of in vitro readthrough relative to the controls at levels similar to those in the stable cell reporter assays. These results indicate that Ataluren modulates termination efficiency at premature nonsense codons.
Related Articles
AbsorptionNot Available
Volume of distributionNot Available
Protein bindingNot Available
MetabolismNot Available
Route of eliminationNot Available
Half life3-6 hours
ClearanceNot Available
ToxicityNot Available
Affected organisms
  • Humans and other mammals
PathwaysNot Available
Pharmacogenomic Effects/ADRs Not Available
Drug Interactions Not Available
Food InteractionsNot Available
Synthesis ReferenceNot Available
General References
  1. Welch EM, Barton ER, Zhuo J, Tomizawa Y, Friesen WJ, Trifillis P, Paushkin S, Patel M, Trotta CR, Hwang S, Wilde RG, Karp G, Takasugi J, Chen G, Jones S, Ren H, Moon YC, Corson D, Turpoff AA, Campbell JA, Conn MM, Khan A, Almstead NG, Hedrick J, Mollin A, Risher N, Weetall M, Yeh S, Branstrom AA, Colacino JM, Babiak J, Ju WD, Hirawat S, Northcutt VJ, Miller LL, Spatrick P, He F, Kawana M, Feng H, Jacobson A, Peltz SW, Sweeney HL: PTC124 targets genetic disorders caused by nonsense mutations. Nature. 2007 May 3;447(7140):87-91. Epub 2007 Apr 22. [PubMed:17450125 ]
  2. Hirawat S, Welch EM, Elfring GL, Northcutt VJ, Paushkin S, Hwang S, Leonard EM, Almstead NG, Ju W, Peltz SW, Miller LL: Safety, tolerability, and pharmacokinetics of PTC124, a nonaminoglycoside nonsense mutation suppressor, following single- and multiple-dose administration to healthy male and female adult volunteers. J Clin Pharmacol. 2007 Apr;47(4):430-44. [PubMed:17389552 ]
  3. Hamed SA: Drug evaluation: PTC-124--a potential treatment of cystic fibrosis and Duchenne muscular dystrophy. IDrugs. 2006 Nov;9(11):783-9. [PubMed:17096300 ]
External Links
ATC CodesM09AX03
AHFS CodesNot Available
PDB EntriesNot Available
FDA labelNot Available
MSDSNot Available
Clinical Trials
Clinical Trials
1Not Yet RecruitingTreatmentHealthy Volunteers1
2Active Not RecruitingTreatmentDuchenne's Muscular Dystrophy (DMD)1
2CompletedTreatmentCystic Fibrosis (CF)4
2CompletedTreatmentDuchenne's Muscular Dystrophy (DMD)1
2SuspendedTreatmentAmino Acid Metabolism, Inborn Errors1
2SuspendedTreatmentHemophilia A / Hereditary factor IX deficiency1
2TerminatedTreatmentBecker's Muscular Dystrophy (BMD) / Duchenne's Muscular Dystrophy (DMD)1
2TerminatedTreatmentDuchenne's Muscular Dystrophy (DMD)1
2, 3CompletedTreatmentBecker's Muscular Dystrophy (BMD) / Duchenne's Muscular Dystrophy (DMD)1
2, 3TerminatedTreatmentBecker's Muscular Dystrophy (BMD) / Duchenne's Muscular Dystrophy (DMD)1
3Active Not RecruitingTreatmentBecker's Muscular Dystrophy (BMD) / Duchenne's Muscular Dystrophy (DMD) / Dystrophinopathies1
3Active Not RecruitingTreatmentCystic Fibrosis (CF)2
3CompletedTreatmentCystic Fibrosis (CF)2
3CompletedTreatmentGenetic Diseases, Inborn / Genetic Diseases, X-Linked / Muscular Diseases / Muscular Disorders, Atrophic / Muscular Dystrophy / Muscular Dystrophy, Duchenne / Musculoskeletal Disorders / Nervous System Diseases / Neuromuscular Diseases1
3Enrolling by InvitationTreatmentBecker's Muscular Dystrophy (BMD) / Duchenne's Muscular Dystrophy (DMD)1
3Enrolling by InvitationTreatmentCystic Fibrosis (CF)1
3Enrolling by InvitationTreatmentGenetic Diseases, Inborn / Genetic Diseases, X-Linked / Muscular Diseases / Muscular Disorders, Atrophic / Muscular Dystrophy / Muscular Dystrophy, Duchenne / Musculoskeletal Disorders / Nervous System Diseases / Neuromuscular Diseases1
Not AvailableRecruitingNot AvailableMuscular Dystrophy, Duchenne1
ManufacturersNot Available
PackagersNot Available
Dosage forms
GranuleOral1000 mg
GranuleOral125 mg
GranuleOral250 mg
PricesNot Available
PatentsNot Available
Experimental PropertiesNot Available
Predicted Properties
Water Solubility0.117 mg/mLALOGPS
pKa (Strongest Acidic)3.9ChemAxon
pKa (Strongest Basic)-1.6ChemAxon
Physiological Charge-1ChemAxon
Hydrogen Acceptor Count4ChemAxon
Hydrogen Donor Count1ChemAxon
Polar Surface Area76.22 Å2ChemAxon
Rotatable Bond Count3ChemAxon
Refractivity94.66 m3·mol-1ChemAxon
Polarizability27.66 Å3ChemAxon
Number of Rings3ChemAxon
Rule of FiveYesChemAxon
Ghose FilterYesChemAxon
Veber's RuleYesChemAxon
MDDR-like RuleYesChemAxon
Predicted ADMET features
Human Intestinal Absorption+1.0
Blood Brain Barrier+0.9681
Caco-2 permeable-0.5391
P-glycoprotein substrateNon-substrate0.8144
P-glycoprotein inhibitor INon-inhibitor0.8905
P-glycoprotein inhibitor IINon-inhibitor0.9784
Renal organic cation transporterNon-inhibitor0.9401
CYP450 2C9 substrateNon-substrate0.8214
CYP450 2D6 substrateNon-substrate0.8367
CYP450 3A4 substrateNon-substrate0.661
CYP450 1A2 substrateInhibitor0.7977
CYP450 2C9 inhibitorNon-inhibitor0.7856
CYP450 2D6 inhibitorNon-inhibitor0.885
CYP450 2C19 inhibitorInhibitor0.5857
CYP450 3A4 inhibitorNon-inhibitor0.8332
CYP450 inhibitory promiscuityLow CYP Inhibitory Promiscuity0.5533
Ames testNon AMES toxic0.7057
BiodegradationNot ready biodegradable0.9828
Rat acute toxicity2.3907 LD50, mol/kg Not applicable
hERG inhibition (predictor I)Weak inhibitor0.997
hERG inhibition (predictor II)Non-inhibitor0.8984
ADMET data is predicted using admetSAR, a free tool for evaluating chemical ADMET properties. (23092397 )
Mass Spec (NIST)Not Available
Spectrum TypeDescriptionSplash Key
Predicted LC-MS/MSPredicted LC-MS/MS Spectrum - 10V, PositiveNot Available
Predicted LC-MS/MSPredicted LC-MS/MS Spectrum - 20V, PositiveNot Available
Predicted LC-MS/MSPredicted LC-MS/MS Spectrum - 40V, PositiveNot Available
Predicted LC-MS/MSPredicted LC-MS/MS Spectrum - 10V, NegativeNot Available
Predicted LC-MS/MSPredicted LC-MS/MS Spectrum - 20V, NegativeNot Available
Predicted LC-MS/MSPredicted LC-MS/MS Spectrum - 40V, NegativeNot Available
DescriptionThis compound belongs to the class of chemical entities known as phenyloxadiazoles. These are polycyclic aromatic compounds containing a benzene ring linked to a 1,2,4-oxadiazole ring through a CC or CN bond.
KingdomChemical entities
Super ClassOrganic compounds
ClassOrganoheterocyclic compounds
Sub ClassAzoles
Direct ParentPhenyloxadiazoles
Alternative Parents
  • Phenyl-1,2,4-oxadiazole
  • Benzoic acid or derivatives
  • Benzoic acid
  • Benzoyl
  • Fluorobenzene
  • Halobenzene
  • Aryl fluoride
  • Aryl halide
  • Monocyclic benzene moiety
  • Benzenoid
  • Heteroaromatic compound
  • Monocarboxylic acid or derivatives
  • Azacycle
  • Oxacycle
  • Carboxylic acid
  • Carboxylic acid derivative
  • Organonitrogen compound
  • Organofluoride
  • Organohalogen compound
  • Hydrocarbon derivative
  • Organic oxide
  • Organopnictogen compound
  • Organic oxygen compound
  • Organic nitrogen compound
  • Organooxygen compound
  • Aromatic heteromonocyclic compound
Molecular FrameworkAromatic heteromonocyclic compounds
External DescriptorsNot Available
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Drug created on October 21, 2007 16:23 / Updated on August 17, 2016 12:24