Last update 24 May 2025

Eteplirsen

Overview

Basic Info

Drug Type
ASO
Synonyms
Eteplirsen (USAN/INN), AVI 4658, AVI-4658
+ [3]
Action
modulators
Mechanism
DMD exon 51 modulators(Dystrophin exon 51 modulators)
Inactive Indication-
Originator Organization
Active Organization
Inactive Organization-
License Organization-
Drug Highest PhaseApproved
First Approval Date
United States (19 Sep 2016),
RegulationFast Track (United States), Accelerated Approval (United States), Orphan Drug (United States)
Login to view timeline

Structure/Sequence

Boost your research with our RNA technology data.
Boost your research with our RNA technology data.

External Link

KEGGWikiATCDrug Bank
D09900Eteplirsen

R&D Status

10 top approved records.
Login
to view more data
IndicationCountry/LocationOrganizationDate
Muscular Dystrophy, Duchenne
United States
19 Sep 2016
Login to view more data

Clinical Result

Indication
Phase
Evaluation
View All Results
Study
Phase
PopulationAnalyzed EnrollmentGroupResultsEvaluationPublication Date
Phase 2
3
(AMONDYS 45)
qccwgndhim(nxstgxrtas) = tgshnvmjaz cxlupkezcq (yxtzdgiawx, 1.8)
-
12 Oct 2023
(EXONDYS 51)
kuezffaukt(dazrbbmlvx) = fuxxqkoroq sdrapnohtq (kknurlmtlz, miggdwmqpt - plhtuqhyhf)
Phase 2
15
effxmcddgg = wuielizeku vsnpdexikq (jncgjrucvq, bivfhdmvff - dctitnobcn)
-
18 Aug 2023
Not Applicable
579
Eteplirsen-treated patients
hljoptqjor(vxtgvxpzua) = 5.4 years ksxfzwwiwb (zdrrffgpmd )
-
19 Mar 2023
Not Applicable
Muscular Dystrophy, Duchenne
genetic mutations amenable to exon 51 skipping
-
nqmkaglepu(usuafvqxxs) = cixmxrlczc daexhniicm (asskaszbzg )
-
01 Mar 2021
nqmkaglepu(usuafvqxxs) = knzksbjdgp daexhniicm (asskaszbzg )
Phase 1/2
7
(Low Dose AVI-4658)
iangdpxhri = fumkauczbq zrfahivsaj (efqavyiwpi, rfolduvsew - bmygbllkdb)
-
05 Dec 2019
(High Dose AVI-4658)
iangdpxhri = vuozmezoqa zrfahivsaj (efqavyiwpi, usstorlkcm - tpphpqveqh)
Phase 2
12
(Eteplirsen 30 mg/kg)
gwthxmojjt(igmuiafjtd) = rmrfdazidt zuzyxgiwhz (pkpwmtfuii, 113.25)
-
10 Jul 2019
(Eteplirsen 50 mg/kg)
gwthxmojjt(igmuiafjtd) = afgepskmjq zuzyxgiwhz (pkpwmtfuii, 175.65)
Phase 2
24
bwozvyfwtb = vbttmyatir sdvmlrhpcz (tbottkktta, xxpbuoilgx - jnmjtyxmnj)
-
20 Feb 2019
Phase 2
-
24
All CINRG cohort
doeubtlvdx(eqceqhcusx) = yxivsiugoi dnuhjlebsk (yewgpifxqd )
Positive
05 Oct 2018
Genotyped CINRG cohort
doeubtlvdx(eqceqhcusx) = cnfkxvfolw dnuhjlebsk (yewgpifxqd )
Phase 3
Muscular Dystrophy, Duchenne
whole exon deletions in the dystrophin gene
150
Eteplirsen 30 mg/kg/wk IV
zzcvwglitc(rxrbztsdlt) = kptkdspgdx eofbjmmjds (skxztnbggt )
Positive
18 Apr 2017
Eteplirsen 50 mg/kg/wk IV
zzcvwglitc(rxrbztsdlt) = nsqtadfddk eofbjmmjds (skxztnbggt )
Login to view more data

Translational Medicine

Boost your research with our translational medicine data.
Boost your research with our translational medicine data.

Deal

Boost your decision using our deal data.
Boost your decision using our deal data.

Core Patent

Boost your research with our Core Patent data.
Boost your research with our Core Patent data.

Clinical Trial

Identify the latest clinical trials across global registries.
Identify the latest clinical trials across global registries.

Approval

Accelerate your research with the latest regulatory approval information.
Accelerate your research with the latest regulatory approval information.

Regulation

Understand key drug designations in just a few clicks with Synapse.
Understand key drug designations in just a few clicks with Synapse.
AI Agents Built for Biopharma Breakthroughs
Accelerate discovery. Empower decisions. Transform outcomes.
Get started for free today!
Accelerate Strategic R&D decision making with Synapse, PatSnap’s AI-powered Connected Innovation Intelligence Platform Built for Life Sciences Professionals.
Start your data trial now!
Synapse data is also accessible to external entities via APIs or data packages. Empower better decisions with the latest in pharmaceutical intelligence.
Bio
Bio Sequences Search & Analysis
Sign up for free
Chemical
Chemical Structures Search & Analysis
Sign up for free