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Accession NumberDB05016
TypeSmall Molecule

Ataluren 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 Identifiers
  • PTC124
Approved Prescription ProductsNot Available
Approved Generic Prescription ProductsNot Available
Approved Over the Counter ProductsNot Available
Unapproved/Other Products Not Available
International Brands
TranslarnaPTC Therapeutics
Brand mixturesNot Available
SaltsNot 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
DescriptionThis compound belongs to the class of organic compounds known as benzoic acids. These are organic Compounds containing a benzene ring which bears at least one carboxyl group.
KingdomOrganic compounds
Super ClassBenzenoids
ClassBenzene and substituted derivatives
Sub ClassBenzoic acids and derivatives
Direct ParentBenzoic acids
Alternative Parents
  • Benzoic acid
  • Benzoyl
  • Halobenzene
  • Fluorobenzene
  • Aryl halide
  • Aryl fluoride
  • Heteroaromatic compound
  • Oxadiazole
  • Azole
  • 1,2,4-oxadiazole
  • Oxacycle
  • Azacycle
  • Organoheterocyclic compound
  • Monocarboxylic acid or derivatives
  • Carboxylic acid
  • Carboxylic acid derivative
  • Hydrocarbon derivative
  • Organooxygen compound
  • Organonitrogen compound
  • Organofluoride
  • Organohalogen compound
  • Carbonyl group
  • Aromatic heteromonocyclic compound
Molecular FrameworkAromatic heteromonocyclic compounds
External DescriptorsNot Available
IndicationFor the treatment of cystic fibrosis and muscular dystrophy.
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
SNP Mediated EffectsNot Available
SNP Mediated Adverse Drug ReactionsNot Available
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 )
ManufacturersNot Available
PackagersNot Available
Dosage formsNot Available
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
Mass Spec (NIST)Not Available
SpectraNot 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
Drug InteractionsNot Available
Food InteractionsNot Available
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Drug created on October 21, 2007 16:23 / Updated on February 25, 2016 06:34