Synopsis
Synopsis
0
USDMF
0
CEP/COS
0
JDMF
0
EU WC
0
KDMF
0
VMF
0
FDA Orange Book
0
Canada
0
Australia
0
South Africa
0
Listed Dossiers
DRUG PRODUCT COMPOSITIONS
0
EDQM
0
USP
0
JP
0
Others
0
US Patents
0
US Exclusivities
0
Health Canada Patents
0
Data Compilation #PharmaFlow
0
Stock Recap #PipelineProspector
0
Weekly News Recap #Phispers
US Medicaid
NA
Annual Reports
NA
Finished Drug Prices
NA
1. Ptc 124
2. Ptc-124
3. Ptc124
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
| Molecular Weight | 284.24 g/mol |
|---|---|
| Molecular Formula | C15H9FN2O3 |
| 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 Ų |
| 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 |
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
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
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.
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.
Ataluren plasma half-life ranges from 2-6 hours and is unaffected either by dose or repeated administration.
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.
API SUPPLIERS
Lifecare Labs has been developing innovative products in specialist areas of medicine.
FULL SCREEN VIEW Click here to open all results in a new tab [this preview display 10 results]
NDC API
NDC Package Code : 48087-0138
Start Marketing Date : 2018-11-29
End Marketing Date : 2024-12-31
Dosage Form (Strength) : POWDER (1kg/kg)
Marketing Category : BULK INGREDIENT
FULL SCREEN VIEW Click here to open all results in a new tab [this preview display 10 results]Listed Suppliers
Lifecare Labs has been developing innovative products in specialist areas of medicine.
About the Company : Lifecare Labs specializes in producing bulk drugs (APIs) and intermediates that adhere to the industry's stringent quality parameters. Its objective is to deliver excellence throug...
FULL SCREEN VIEW Click here to open all results in a new tab [this preview display 10 results]API Reference Price
API Imports and Exports
| Importing Country | Total Quantity (KGS) |
Average Price (USD/KGS) |
Number of Transactions |
|---|
Upgrade, download data, analyse, strategize, subscribe with us 
Drugs in Development
Details:
Translarna (ataluren) is a dystrophin expression stimulator small molecule drug candidate, indicated for the treatment of nonsense mutated duchenne muscular dystrophy , in patients aged 2 or above.
Lead Product(s): Ataluren
Therapeutic Area: Genetic Disease Brand Name: Translarna
Study Phase: ApprovedProduct Type: Small molecule
Sponsor: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable October 18, 2024
Lead Product(s) : Ataluren
Therapeutic Area : Genetic Disease
Highest Development Status : Approved
Partner/Sponsor/Collaborator : Not Applicable
Deal Size : Not Applicable
Deal Type : Not Applicable
CHMP Maintains Negative Opinion on Translarna™ Reexamination
Details : Translarna (ataluren) is a dystrophin expression stimulator small molecule drug candidate, indicated for the treatment of nonsense mutated duchenne muscular dystrophy , in patients aged 2 or above.
Brand Name : Translarna
Molecule Type : Small molecule
Upfront Cash : Not Applicable
October 18, 2024
Details:
Translarna (ataluren) is a protein restoration therapy under phase 3 development for nonsense mutation Duchenne Muscular Dystrophy, enabling functioning protein formation in patients.
Lead Product(s): Ataluren
Therapeutic Area: Genetic Disease Brand Name: Translarna
Study Phase: Phase IIIProduct Type: Small molecule
Sponsor: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable January 25, 2024
Lead Product(s) : Ataluren
Therapeutic Area : Genetic Disease
Highest Development Status : Phase III
Partner/Sponsor/Collaborator : Not Applicable
Deal Size : Not Applicable
Deal Type : Not Applicable
CHMP Issues Negative Opinion On Translarna™ Renewal
Details : Translarna (ataluren) is a protein restoration therapy under phase 3 development for nonsense mutation Duchenne Muscular Dystrophy, enabling functioning protein formation in patients.
Brand Name : Translarna
Molecule Type : Small molecule
Upfront Cash : Not Applicable
January 25, 2024
Details:
Translarna (ataluren) is a dystrophin expression stimulator small molecule drug candidate, which is currently being evaluated for the treatment of duchenne muscular dystrophy from a nonsense mutated dystrophin gene, in patients aged 2 or above via oral suspension.
Lead Product(s): Ataluren
Therapeutic Area: Genetic Disease Brand Name: Translarna
Study Phase: ApprovedProduct Type: Small molecule
Sponsor: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable December 05, 2023
Lead Product(s) : Ataluren
Therapeutic Area : Genetic Disease
Highest Development Status : Approved
Partner/Sponsor/Collaborator : Not Applicable
Deal Size : Not Applicable
Deal Type : Not Applicable
PTC Therapeutics Provides Updates on Translarna Regulatory Activities
Details : Translarna (ataluren) is a dystrophin expression stimulator small molecule drug candidate, which is currently being evaluated for the treatment of duchenne muscular dystrophy from a nonsense mutated dystrophin gene, in patients aged 2 or above via oral s...
Brand Name : Translarna
Molecule Type : Small molecule
Upfront Cash : Not Applicable
December 05, 2023
Details:
The strategic financing will support the acceleration of PTC’s robust and diversified pipeline having lead asset Translarna (ataluren), business development opportunities and general corporate purposes.
Lead Product(s): Ataluren
Therapeutic Area: Genetic Disease Brand Name: Translarna
Study Phase: ApprovedProduct Type: Small molecule
Sponsor: Blackstone Life Sciences
Deal Size: $50.0 million Upfront Cash: Undisclosed
Deal Type: Collaboration October 27, 2022
Lead Product(s) : Ataluren
Therapeutic Area : Genetic Disease
Highest Development Status : Approved
Partner/Sponsor/Collaborator : Blackstone Life Sciences
Deal Size : $50.0 million
Deal Type : Collaboration
Details : The strategic financing will support the acceleration of PTC’s robust and diversified pipeline having lead asset Translarna (ataluren), business development opportunities and general corporate purposes.
Brand Name : Translarna
Molecule Type : Small molecule
Upfront Cash : Undisclosed
October 27, 2022
Details:
Translarna (ataluren), discovered and developed by PTC Therapeutics, is a protein restoration therapy designed to enable the formation of a functioning protein in patients with genetic disorders caused by a nonsense mutation.
Lead Product(s): Ataluren
Therapeutic Area: Genetic Disease Brand Name: Translarna
Study Phase: ApprovedProduct Type: Small molecule
Sponsor: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable October 11, 2022
Lead Product(s) : Ataluren
Therapeutic Area : Genetic Disease
Highest Development Status : Approved
Partner/Sponsor/Collaborator : Not Applicable
Deal Size : Not Applicable
Deal Type : Not Applicable
PTC Therapeutics to Present Clinical and Real-World Evidence on Translarna™ at the Annual Congre...
Details : Translarna (ataluren), discovered and developed by PTC Therapeutics, is a protein restoration therapy designed to enable the formation of a functioning protein in patients with genetic disorders caused by a nonsense mutation.
Brand Name : Translarna
Molecule Type : Small molecule
Upfront Cash : Not Applicable
October 11, 2022
Details:
Translarna (ataluren) is the first licensed treatment for DMD that addresses the loss of dystrophin. It works by allowing the body to read over the mutation in the DNA and continue to produce dystrophin.
Lead Product(s): Ataluren
Therapeutic Area: Genetic Disease Brand Name: Translarna
Study Phase: Phase IIIProduct Type: Small molecule
Sponsor: NICE
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable September 30, 2022
Lead Product(s) : Ataluren
Therapeutic Area : Genetic Disease
Highest Development Status : Phase III
Partner/Sponsor/Collaborator : NICE
Deal Size : Not Applicable
Deal Type : Not Applicable
Details : Translarna (ataluren) is the first licensed treatment for DMD that addresses the loss of dystrophin. It works by allowing the body to read over the mutation in the DNA and continue to produce dystrophin.
Brand Name : Translarna
Molecule Type : Small molecule
Upfront Cash : Not Applicable
September 30, 2022
Details:
Translarna (ataluren), discovered and developed by PTC Therapeutics, Inc., is a protein restoration therapy designed to enable the formation of a functioning protein in patients with genetic disorders caused by a nonsense mutation.
Lead Product(s): Ataluren
Therapeutic Area: Genetic Disease Brand Name: Translarna
Study Phase: Phase IIIProduct Type: Small molecule
Sponsor: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable June 20, 2022
Lead Product(s) : Ataluren
Therapeutic Area : Genetic Disease
Highest Development Status : Phase III
Partner/Sponsor/Collaborator : Not Applicable
Deal Size : Not Applicable
Deal Type : Not Applicable
PTC Therapeutics to Host Call to Review Topline Results from Study 041 for Translarna™ (ataluren...
Details : Translarna (ataluren), discovered and developed by PTC Therapeutics, Inc., is a protein restoration therapy designed to enable the formation of a functioning protein in patients with genetic disorders caused by a nonsense mutation.
Brand Name : Translarna
Molecule Type : Small molecule
Upfront Cash : Not Applicable
June 20, 2022
Details:
Translarna is the only treatment for the underlying cause of Duchenne caused by a nonsense mutation. Clinical trials and real-world evidence have demonstrated Translarna's potential to slow disease progression and improve patient outcomes.
Lead Product(s): Ataluren
Therapeutic Area: Genetic Disease Brand Name: Translarna
Study Phase: Phase IIProduct Type: Small molecule
Sponsor: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable October 25, 2021
Lead Product(s) : Ataluren
Therapeutic Area : Genetic Disease
Highest Development Status : Phase II
Partner/Sponsor/Collaborator : Not Applicable
Deal Size : Not Applicable
Deal Type : Not Applicable
PTC Announces Expansion of Translarna™ Label to Include Ambulatory Patients as Young as 2 Years ...
Details : Translarna is the only treatment for the underlying cause of Duchenne caused by a nonsense mutation. Clinical trials and real-world evidence have demonstrated Translarna's potential to slow disease progression and improve patient outcomes.
Brand Name : Translarna
Molecule Type : Small molecule
Upfront Cash : Not Applicable
October 25, 2021
Details:
Real-world results from the STRIDE patient registry demonstrating that treatment with Translarna™ (ataluren) delays loss of ambulation by more than five years in boys with nonsense mutation Duchenne muscular dystrophy (nmDMD) compared to standard of care (SoC) alone.
Lead Product(s): Ataluren
Therapeutic Area: Genetic Disease Brand Name: Translarna
Study Phase: Phase IIProduct Type: Small molecule
Sponsor: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable September 20, 2021
Lead Product(s) : Ataluren
Therapeutic Area : Genetic Disease
Highest Development Status : Phase II
Partner/Sponsor/Collaborator : Not Applicable
Deal Size : Not Applicable
Deal Type : Not Applicable
STRIDE Data Show Translarna™ Delays Loss of Ambulation by More Than Five Years in Boys with Nons...
Details : Real-world results from the STRIDE patient registry demonstrating that treatment with Translarna™ (ataluren) delays loss of ambulation by more than five years in boys with nonsense mutation Duchenne muscular dystrophy (nmDMD) compared to standard of ca...
Brand Name : Translarna
Molecule Type : Small molecule
Upfront Cash : Not Applicable
September 20, 2021
Details:
PTC Therapeutics is planning to hold talks with the US FDA on approval after the latest Phase II Study 045 of its drug, Translarna (ataluren), failed to significantly improve dystrophin expression in patients with nonsense mutation Duchenne muscular dystrophy.
Lead Product(s): Ataluren
Therapeutic Area: Genetic Disease Brand Name: Translarna
Study Phase: Phase IIProduct Type: Small molecule
Sponsor: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable February 04, 2021
Lead Product(s) : Ataluren
Therapeutic Area : Genetic Disease
Highest Development Status : Phase II
Partner/Sponsor/Collaborator : Not Applicable
Deal Size : Not Applicable
Deal Type : Not Applicable
Details : PTC Therapeutics is planning to hold talks with the US FDA on approval after the latest Phase II Study 045 of its drug, Translarna (ataluren), failed to significantly improve dystrophin expression in patients with nonsense mutation Duchenne muscular dyst...
Brand Name : Translarna
Molecule Type : Small molecule
Upfront Cash : Not Applicable
February 04, 2021
FULL SCREEN VIEW Click here to open all results in a new tab [this preview display 10 results]FDF Dossiers
Regulatory Info :
Registration Country : Sweden
Brand Name : trans couplers
Dosage Form : GRANULES FOR ORAL SUSPENSION
Dosage Strength : 125 MG
Packaging :
Approval Date :
Application Number :
Regulatory Info :
Registration Country : Sweden
Portfolio PDF
Product Web Link
Virtual Booth
Digital Content
Website
Corporate PDF
Regulatory Info :
Registration Country : Sweden
Brand Name : trans couplers
Dosage Form : GRANULES FOR ORAL SUSPENSION
Dosage Strength : 250 MG
Packaging :
Approval Date :
Application Number :
Regulatory Info :
Registration Country : Sweden
Portfolio PDF
Product Web Link
Virtual Booth
Digital Content
Website
Corporate PDF
Regulatory Info :
Registration Country : Sweden
Brand Name : trans couplers
Dosage Form : GRANULES FOR ORAL SUSPENSION
Dosage Strength : 1000 MG
Packaging :
Approval Date :
Application Number :
Regulatory Info :
Registration Country : Sweden
Portfolio PDF
Product Web Link
Virtual Booth
Digital Content
Website
Corporate PDF
FULL SCREEN VIEW Click here to open all results in a new tab [this preview display 10 results]Europe
Regulatory Info :
Registration Country : Sweden
Brand Name : trans couplers
Dosage Form : GRANULES FOR ORAL SUSPENSION
Dosage Strength : 125 MG
Packaging :
Approval Date :
Application Number :
Regulatory Info :
Registration Country : Sweden
Portfolio PDF
Product Web Link
Virtual Booth
Digital Content
Website
Corporate PDF
Regulatory Info :
Registration Country : Sweden
Brand Name : trans couplers
Dosage Form : GRANULES FOR ORAL SUSPENSION
Dosage Strength : 250 MG
Packaging :
Approval Date :
Application Number :
Regulatory Info :
Registration Country : Sweden
Portfolio PDF
Product Web Link
Virtual Booth
Digital Content
Website
Corporate PDF
Regulatory Info :
Registration Country : Sweden
Brand Name : trans couplers
Dosage Form : GRANULES FOR ORAL SUSPENSION
Dosage Strength : 1000 MG
Packaging :
Approval Date :
Application Number :
Regulatory Info :
Registration Country : Sweden
Portfolio PDF
Product Web Link
Virtual Booth
Digital Content
Website
Corporate PDF
FULL SCREEN VIEW Click here to open all results in a new tab [this preview display 10 results]Digital Content
NEWS #PharmaBuzz
18 Oct 2024
// PR NEWSWIRE
https://www.prnewswire.com/news-releases/chmp-maintains-negative-opinion-on-translarna-reexamination-302279958.html
08 Aug 2024
// PR NEWSWIRE
https://www.prnewswire.com/news-releases/ptc-therapeutics-provides-corporate-update-and-reports-second-quarter-2024-financial-results-302218200.html
12 Jul 2024
// PHARMABIZ
28 Jun 2024
// PR NEWSWIRE
https://www.prnewswire.com/news-releases/chmp-issues-negative-opinion-on-translarna-following-european-commission-request-for-review-302185263.html
25 Jan 2024
// FIERCE PHARMA
https://www.fiercepharma.com/pharma/ptcs-re-examination-bid-dmd-drug-translarna-falls-short-europe
15 Sep 2023
// EMA
https://www.ema.europa.eu/en/news/ema-recommends-non-renewal-authorisation-duchenne-muscular-dystrophy-medicine-translarna
Market Place
APIs
Reply
17 Jul 2023
Reply
08 Jun 2023
Reply
30 May 2022
FULL SCREEN VIEW Click here to open all results in a new tab [this preview display 10 results]
ABOUT THIS PAGE
LOOKING FOR A SUPPLIER?
Share 
Comment (5)
