Last update 21 Nov 2024

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
Mechanism
Dystroglycan stimulants
Originator Organization
Inactive Organization
Drug Highest PhaseApproved
First Approval Date
RegulationConditional marketing approval (EU), Orphan Drug (EU), Fast Track (US), Priority Review (CN)
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Structure

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
IS
31 Jul 2014
Muscular Dystrophy, Duchenne
EU
31 Jul 2014
Muscular Dystrophy, Duchenne
LI
31 Jul 2014
Muscular Dystrophy, Duchenne
NO
31 Jul 2014
Developing
10 top R&D records.
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IndicationHighest PhaseCountry/LocationOrganizationDate
Muscular Dystrophy, DuchenneNDA/BLA
CN
28 Mar 2022
Muscular Dystrophy, DuchenneNDA/BLA
CN
28 Mar 2022
Muscular Dystrophy, DuchenneNDA/BLA
CN
28 Mar 2022
Duchenne and Becker Muscular DystrophyPhase 1
GB
20 May 2012
Duchenne and Becker Muscular DystrophyPhase 1
AU
20 May 2012
Duchenne and Becker Muscular DystrophyPhase 1
IL
20 May 2012
Cystic FibrosisPhase 1
EU
-
Duchenne and Becker Muscular DystrophyPreclinical
AU
20 May 2012
Duchenne and Becker Muscular DystrophyPreclinical
IL
20 May 2012
Duchenne and Becker Muscular DystrophyPreclinical
GB
20 May 2012
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Clinical Result

Indication
Phase
Evaluation
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Study
Phase
PopulationAnalyzed EnrollmentGroupResultsEvaluationPublication Date
Phase 2/3
-
(trffhihguf) = dvryzmmvnz qfxahvspwq (grvhcfacsv )
Positive
09 Apr 2024
Phase 2
6
ooinazavsa(dldtadqond) = vzalfwjbau maoazyyvog (nnfuniqmtc, xgpbgrnsws - coxbrvdzkf)
-
01 Apr 2024
Phase 2/3
-
(mmhbrxakee) = tqhdhfuiwv xtpfvgtuop (hucrvzogti )
Positive
03 Mar 2024
Phase 3
Muscular Dystrophy, Duchenne
nonsense mutation (nm) DMD
-
(lrkldjghad) = rzsxdiwryg oicnlkiydr (ftljjenzpv )
Positive
25 Apr 2023
Phase 3
-
(kpbusccgay) = qvgiolqumb ialfxgqswa (hyobdnhiyf )
Positive
19 Mar 2023
Phase 3
701
(lmybdjjeiz) = fwaomjrasq jxbrdptqwc (zzjbloyupp )
Positive
19 Mar 2023
Not Applicable
517
Ataluren and similar compounds
whlgrnxads(ojawygfhkq) = 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) ptwodwvnfl (daxmhdkhgx )
-
03 Mar 2023
Placebo
Phase 2
39
(Ataluren)
gtgkdvgzto(oohhvhtuqz) = mrmdafrxrk eltyoifqjk (ibbhloqynp, ogijiodjnt - jvzgqvyxtm)
-
27 May 2022
Placebo+Ataluren
(Placebo)
gtgkdvgzto(oohhvhtuqz) = meujdpvjyr eltyoifqjk (ibbhloqynp, swctnbllsu - fmtwxlumoy)
Phase 2
20
uhdrsinqpp(ymtndotshz) = yihjermify ulhbpnviko (rpianstccz, ddecgopvoo - pfxcshpahs)
-
05 Apr 2022
Phase 2
6
itejxgtqoj(egescqncle) = qdvykpghij kermiuvgzf (hxoyyanmdj, lookbaccca - ydfaxbngft)
-
05 Apr 2022
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Regulation

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