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Ataluren

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Chemistry

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Also known as: 775304-57-9, Ptc124, 3-(5-(2-fluorophenyl)-1,2,4-oxadiazol-3-yl)benzoic acid, 3-[5-(2-fluorophenyl)-1,2,4-oxadiazol-3-yl]benzoic acid, Ptc-124, Ataluren (ptc124)

Molecular Formula

C₁₅H₉FN₂O₃

Molecular Weight

284.24 g/mol

InChI Key

OOUGLTULBSNHNF-UHFFFAOYSA-N

FDA UNII

K16AME9I3V

Ataluren 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 patients whose disease is caused by a specific genetic defect (called a nonsense mutation) in the dystrophin gene. This drug does not yet have approval by the US Food and Drug Administration or by Health Canada for any indications.

1 2D Structure

Ataluren

2 Identification

2.1 Computed Descriptors

2.1.1 IUPAC Name

3-[5-(2-fluorophenyl)-1,2,4-oxadiazol-3-yl]benzoic acid

2.1.2 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)

2.1.3 InChI Key

OOUGLTULBSNHNF-UHFFFAOYSA-N

2.1.4 Canonical SMILES

C1=CC=C(C(=C1)C2=NC(=NO2)C3=CC(=CC=C3)C(=O)O)F

2.2 Other Identifiers

2.2.1 UNII

K16AME9I3V

2.3 Synonyms

2.3.1 MeSH Synonyms

1. Ptc 124

2. Ptc-124

3. Ptc124

2.3.2 Depositor-Supplied Synonyms

1. 775304-57-9

2. Ptc124

3. 3-(5-(2-fluorophenyl)-1,2,4-oxadiazol-3-yl)benzoic Acid

4. 3-[5-(2-fluorophenyl)-1,2,4-oxadiazol-3-yl]benzoic Acid

5. Ptc-124

6. Ataluren (ptc124)

7. Translarna

8. Ptc124 (ataluren)

9. Ptc 124

10. Benzoic Acid, 3-[5-(2-fluorophenyl)-1,2,4-oxadiazol-3-yl]-

11. K16ame9i3v

12. Ncgc00168759-02

13. Benzoic Acid, 3-(5-(2-fluorophenyl)-1,2,4-oxadiazol-3-yl)-

14. Dsstox_cid_26776

15. Dsstox_rid_81895

16. Dsstox_gsid_46776

17. Ataluren [usan]

18. Cas-775304-57-9

19. Ataluren [usan:inn]

20. Unii-k16ame9i3v

21. Translarna (tn)

22. Ptc124,ataluren

23. Ataluren(ptc124)

24. Ataluren; Ptc124

25. Ptc124 - Ataluren

26. Ataluren (usan/inn)

27. Ataluren [inn]

28. Ataluren [mi]

29. Ec-000.2051

30. Ataluren [who-dd]

31. Schembl60614

32. Mls006011160

33. Chembl256997

34. Gtpl7341

35. Dtxsid5046776

36. Chebi:94805

37. Ex-a385

38. Bcpp000097

39. Hms3656h20

40. Albb-036381

41. Bcp01756

42. Tox21_112631

43. Bbl102158

44. Mfcd09864996

45. Ncgc00168759

46. S6003

47. Stl555957

48. Zinc13831791

49. Akos005146455

50. Tox21_112631_1

51. Ccg-267286

52. Cs-0503

53. Db05016

54. Ex-3387

55. Gs-3946

56. Sb16685

57. Ncgc00168759-04

58. Ncgc00168759-05

59. Ncgc00168759-10

60. Ac-28390

61. Am808091

62. Hy-14832

63. Smr004702931

64. Bb 0261439

65. Ft-0651455

66. Sw219696-1

67. D09323

68. 304f579

69. Au-004/43508117

70. Q753330

71. Brd-k94830329-001-01-4

72. 3-[5-(2-fluoro-phenyl)-[1,2,4]oxadiazol-3-yl]-benzoic Acid

73. 3-[5-(2-fluorophenyl)-[1,2,4]oxadiazol-3-yl]benzoic Acid

74. Ptc124;3-[5-(2-fluorophenyl)-1,2,4-oxadiazol-3-yl]benzoic Acid;ptc-124

75. Jbf

2.4 Create Date

2006-10-26

3 Chemical and Physical Properties

Molecular Formula	C₁₅H₉FN₂O₃
Molecular Weight	284.24 g/mol
XLogP3	3.1
Hydrogen Bond Donor Count	1
Hydrogen Bond Acceptor Count	6
Rotatable Bond Count	3
Exact Mass	284.05972032 g/mol
Monoisotopic Mass	284.05972032 g/mol
Topological Polar Surface Area	76.2 Å²
Heavy Atom Count	21
Formal Charge	0
Complexity	382
Isotope Atom Count	0
Defined Atom Stereocenter Count	0
Undefined Atom Stereocenter Count	0
Defined Bond Stereocenter Count	0
Undefined Bond Stereocenter Count	0
Covalently Bonded Unit Count	1

4 Drug and Medication Information

4.1 Drug Indication

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 patients whose disease is caused by a specific genetic defect (called a nonsense mutation) in the dystrophin gene.

Translarna is indicated for the treatment of Duchenne muscular dystrophy resulting from a nonsense mutation in the dystrophin gene, in ambulatory patients aged 2 years and older. Efficacy has not been demonstrated in non-ambulatory patients.

The presence of a nonsense mutation in the dystrophin gene should be determined by genetic testing.

Treatment of cystic fibrosis

Treatment of dystrophinopathy

5 Pharmacology and Biochemistry

5.1 ATC Code

M09AX03

M - Musculo-skeletal system

M09 - Other drugs for disorders of the musculo-skeletal system

M09A - Other drugs for disorders of the musculo-skeletal system

M09AX - Other drugs for disorders of the musculo-skeletal system

M09AX03 - Ataluren

5.2 Absorption, Distribution and Excretion

Absorption

Peak plasma levels of ataluren are attained approximately 1.5 hours after dosing in subjects who received medicinal product within 30 minutes of a meal.

Route of Elimination

After a single oral dose of radiolabeled ataluren, approximately half of the administered radioactive dose is recovered in the faeces and the remainder was recovered in the urine. In the urine, unchanged ataluren and the acyl glucuronide metabolite account for less than 1% and 49%, respectively, of the administered dose.

5.3 Metabolism/Metabolites

Ataluren is metabolized by conjugation via uridine diphosphate glucuronosyltransferase (UGT) enzymes, predominantly UGT1A9 in liver and intestine. In vivo, the only metabolite detected in plasma after oral administration of radio-labelled ataluren was the ataluren-O-1-acyl glucuronide; exposure to this metabolite in humans was approximately 8% of the plasma AUC of ataluren.

5.4 Biological Half-Life

Ataluren plasma half-life ranges from 2-6 hours and is unaffected either by dose or repeated administration.

5.5 Mechanism of Action

Ataluren enables ribosomal readthrough of mRNA containing premature stop codons that otherwise would result in premature termination of protein chains. Use of ataluren allows cellular machinery to bypass nonsense mutations in genetic material, 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.

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