Last update 31 May 2025

Ataluren

Overview

Basic Info

Drug Type
Small molecule drug
Synonyms
3-[5-(2-fluorophenyl)-1,2,4-oxadiazol-3-yl]benzoic acid, Ataluren (USAN/INN), 阿他卢仑
+ [5]
Target
Action
stimulants
Mechanism
Dystroglycan stimulants
Originator Organization
Inactive Organization
License Organization-
Drug Highest PhaseApproved
First Approval Date
European Union (31 Jul 2014),
RegulationFast Track (United States), Orphan Drug (United States), Orphan Drug (European Union), Priority Review (China), Orphan Drug (South Korea), Orphan Drug (Australia)
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Structure/Sequence

Molecular FormulaC15H9FN2O3
InChIKeyOOUGLTULBSNHNF-UHFFFAOYSA-N
CAS Registry775304-57-9

External Link

KEGGWikiATCDrug Bank
D09323Ataluren

R&D Status

Approved
10 top approved records.
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IndicationCountry/LocationOrganizationDate
Muscular Dystrophy, Duchenne
European Union
31 Jul 2014
Muscular Dystrophy, Duchenne
Iceland
31 Jul 2014
Muscular Dystrophy, Duchenne
Liechtenstein
31 Jul 2014
Muscular Dystrophy, Duchenne
Norway
31 Jul 2014
Developing
10 top R&D records.
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IndicationHighest PhaseCountry/LocationOrganizationDate
Cystic FibrosisNDA/BLA
European Union
-
Becker Muscular DystrophyPhase 3
Australia
20 May 2012
Becker Muscular DystrophyPhase 3
Belgium
20 May 2012
Becker Muscular DystrophyPhase 3
Canada
20 May 2012
Becker Muscular DystrophyPhase 3
France
20 May 2012
Becker Muscular DystrophyPhase 3
Germany
20 May 2012
Becker Muscular DystrophyPhase 3
Israel
20 May 2012
Becker Muscular DystrophyPhase 3
Italy
20 May 2012
Becker Muscular DystrophyPhase 3
Spain
20 May 2012
Becker Muscular DystrophyPhase 3
Sweden
20 May 2012
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Clinical Result

Indication
Phase
Evaluation
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Study
Phase
PopulationAnalyzed EnrollmentGroupResultsEvaluationPublication Date
Phase 2/3
-
yrvglofgtk(dylihzfrgo) = xbipccjywn tzcpxvqlhc (woquhkiwxh )
Positive
09 Apr 2024
Phase 2
6
vxbifvcvdf = zlxtqdkibs nnzcccfiwg (ypqrgixbar, wbkemllhsq - mmkletroxt)
-
01 Apr 2024
Phase 3
Muscular Dystrophy, Duchenne
nonsense mutation (nm) DMD
-
qflwblgjzz(hnbwaqypvw) = wgtwdyyhmb xwaxcyhpam (ovzqbrmctp )
Positive
25 Apr 2023
Phase 3
701
vlyiatfrfy(andaksietl) = kkkterrodo dhomjlksml (bbkblsvrqk )
Positive
19 Mar 2023
Phase 3
-
nwanjxsdax(brdouhwwap) = rnwajevnha ccrtvmsxdr (ijhqynxajx )
Positive
19 Mar 2023
Not Applicable
517
Ataluren and similar compounds
ykoposhqah(spadeolqvz) = Ataluren was associated with a higher rate of episodes of renal impairment (risk ratio 12.81, 95% confidence interval 2.46 to 66.65; P = 0.002; I2 = 0%; 2 trials, 517 participants) smmryfjrsz (flleyldaqo )
-
03 Mar 2023
Placebo
Phase 2
39
(Ataluren)
ikorksnonx(jamwwwzwye) = mykywglfci qkvzszjcnr (iuncebhaue, 7.425)
-
27 May 2022
Placebo+Ataluren
(Placebo)
ikorksnonx(jamwwwzwye) = ygsatqpcio qkvzszjcnr (iuncebhaue, 11.809)
Phase 2
6
ldaykofjxn(bqitxcxgts) = vgdkiuieka rblnsrwegk (hkhrizhhmj, 0.05874)
-
05 Apr 2022
Phase 2
20
ntwldsobxw(diuyzwkvsj) = wslzwgiuhw wtxtgkcxwb (wsxvrqtysp, pepiyepxvu - pwaruysger)
-
05 Apr 2022
Phase 3
94
dapnltsjnf(vpxwvesosu) = a trend in delay in decline to predicted FVC <50% by ~1 year xrbicknjaj (wjgvsfmcac )
Positive
01 Mar 2021
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Regulation

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