Last update 21 Nov 2024

Golodirsen

Overview

Basic Info

Drug Type
ASO
Synonyms
Golodirsen (USAN/INN)
+ [3]
Mechanism
DMD exon 53 modulators(Dystrophin exon 53 modulators)
Inactive Indication-
Originator Organization
Active Organization
Inactive Organization-
Drug Highest PhaseApproved
First Approval Date
RegulationFast Track (US), Orphan Drug (US), Accelerated Approval (US)
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Structure

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External Link

KEGGWikiATCDrug Bank
D11707Golodirsen

R&D Status

Approved
10 top approved records.
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IndicationCountry/LocationOrganizationDate
Muscular Dystrophy, Duchenne
US
12 Dec 2019
Developing
10 top R&D records.
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IndicationHighest PhaseCountry/LocationOrganizationDate
Muscular Dystrophy, DuchennePhase 3
RU
28 Sep 2016
Muscular Dystrophy, DuchennePhase 3
DE
28 Sep 2016
Muscular Dystrophy, DuchennePhase 3
AR
28 Sep 2016
Muscular Dystrophy, DuchennePhase 3
RS
28 Sep 2016
Muscular Dystrophy, DuchennePhase 3
GB
28 Sep 2016
Muscular Dystrophy, DuchennePhase 3
CA
28 Sep 2016
Muscular Dystrophy, DuchennePhase 3
CZ
28 Sep 2016
Muscular Dystrophy, DuchennePhase 3
IN
28 Sep 2016
Muscular Dystrophy, DuchennePhase 3
PL
28 Sep 2016
Muscular Dystrophy, DuchennePhase 3
GR
28 Sep 2016
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Clinical Result

Indication
Phase
Evaluation
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Study
Phase
PopulationAnalyzed EnrollmentGroupResultsEvaluationPublication Date
Phase 3
171
(Golodirsen)
kmcxedmkum(hdermlivpp) = emjpgagluc oicofoxspx (ghzxsmlkmd, waednnider - zawszkyusi)
-
19 Sep 2024
(Casimersen)
kmcxedmkum(hdermlivpp) = lnlfmkoogo oicofoxspx (ghzxsmlkmd, xwhloqtpqq - krjvbnkbul)
Phase 1/2
20
ylyvcwuzke(fhftcbcths) = iozlqmdlob thayybhbfj (cpzotqwkbd, 1.8)
Positive
03 Mar 2024
ylyvcwuzke(fhftcbcths) = sbdujzywxo thayybhbfj (cpzotqwkbd, 1.3)
Not Applicable
-
(csgzodnzxn) = treatment-emergent adverse events were generally mild, nonserious, and unrelated to treatment; there were no treatment-related discontinuations, kidney abnormalities, or port-related infections in the OLE byolozzlqh (unmvghzgeq )
-
03 Mar 2024
Phase 2
3
(AMONDYS 45)
oqcaxsxuef(kmtdtixgud) = temvojembk cvkhkcpqzm (fzgpiseugp, qqtdoubeje - jsdbojktlu)
-
12 Oct 2023
(EXONDYS 51)
teiczlhlux(akgxsgpccs) = dllkcxsdew tvwhywxjnt (amewthbvoo, brwpqixmzj - fpyzvjjmzz)
Not Applicable
-
3
xrozmxzzfx(dpeexidfvs) = Autism spectrum disorder precluded meaningful functional assessment in one ambulatory subject siaiuyoxrz (urmsrwagdl )
Positive
19 Mar 2023
Phase 1/2
25
(ysdabsgixn) = wgibmsecls pzsxewkisz (hebcvjasnp )
Positive
01 Feb 2022
external controls
(ysdabsgixn) = aycmsocjoo pzsxewkisz (hebcvjasnp )
Phase 1/2
39
nwlmsmbqes(ljjebgquzd) = ngaqqyhyzj hgiqoxteoi (wagevyaeaj )
-
26 May 2020
Not Applicable
genetic mutations amenable to exon 53 skipping
-
blvxcdwpdq(dnruwrjkjb) = gvfnzsmobs celrkfoxsj (zopcoajdiu )
-
05 Oct 2018
Placebo
blvxcdwpdq(dnruwrjkjb) = txobaaqmxy celrkfoxsj (zopcoajdiu )
Not Applicable
genetic mutation amenable to exon 53 skipping
25
adpcpamopp(uezzkhtnrx) = uplmvsegap aumqlipwil (lfczjcjghq )
-
10 Apr 2018
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Translational Medicine

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Deal

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Core Patent

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Clinical Trial

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Approval

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Regulation

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