eteplirsen

Trade name: Exondys 51

Nucleic acidapprovedOrphan Drug ^FDAFast Track ^FDA

Approved

Sep 19, 2016

Eteplirsen is a medication to treat, but not cure, some types of Duchenne muscular dystrophy (DMD), caused by a specific mutation. Eteplirsen only targets specific mutations and can be used to treat about 14% of DMD cases. Eteplirsen is a form of antisense therapy. — Wikipedia

Clinical trial activity

14 trials · 6 clinical orgs · 1 marketing orgs

Phase 1

Phase 2

Phase 3

Phase 4

Earliest trial started Oct 1, 2007 (NCT00159250)

Timeline

2000s

Jan 1, 2007
Earliest Phase 1 Sponsor(trial)
Oct 23, 2007
Orphan Drug Designation

2010s

Jan 1, 2011
Sarepta Therapeutics — Earliest Phase 2 Sponsor(trial)
Jan 1, 2014
Sarepta Therapeutics — Earliest Phase 3 Sponsor(trial)
Sep 19, 2016
Sarepta Therapeutics — Earliest FDA Approval
Sep 19, 2016
Sarepta Therapeutics — NDA Secondary Org
Sep 19, 2016
Sarepta Therapeutics — NDA Organization

Indications

Approved for

Mechanism of action

Dystrophin pre-mRNAPOSITIVE MODULATOR
Dystrophin pre-mRNA positive modulator
Phosphorodiamidate morpholino oligomer (PMO) that selectively binds to exon 51 of dystrophin pre-mRNA, restoring the open reading frame and ena- bling production of functional truncated in-frame dystrophin protein.
Binds to exon 51 of dystrophin pre-mRNA.
Dystrophin pre-mRNAMODULATOR
Dystrophin pre-mRNA modulator
Phosphorodiamidate morpholino oligomer (PMO) that selectively binds to exon 51 of dystrophin pre-mRNA, restoring the open reading frame and ena- bling production of functional truncated in-frame dystrophin protein.
Binds to exon 51 of dystrophin pre-mRNA.
DystrophinSupplementation
DMD supplementation
- Dystrophin (Dmd) ↗

Approval history

approvedPriority reviewAcceleratedFast trackSep 19, 2016

Chemistry & pharmacology

Chirality: Single Stereoisomer
Inorganic: No
Polymer: No
Delivery: Parenteral
Availability: Prescription Only
Multi-specific: No

Oral

Parenteral

Yes

Topical

First in class

Sources

Also known as

avi 4658
avi-4658
avi-4658
avi-4658
eteplirsen
eteplirsen
eteplirsen
eteplirsen
eteplirsen
eteplirsen
eteplirsen
eteplirsen
exondys 51
exondys 51
exondys 51
(p-deoxy-p-(dimethylamino)](2',3'-dideoxy-2',3'-imino-2',3'-seco)(2'a→5')(c-m5u-c-c-a-a-c-a-m5u-c-a-a-g-g-a-a-g-a-m5u-g-g-c-a-m5u-m5u-m5u-c-m5u-a-g),5'-(p-(4-((2-(2-(2-hydroxyethoxy)ethoxy)ethoxy)carbonyl)-1-piperazinyl)-n,n-dimethylphosphonamidate) rna
srp-5051

Clinical trials

NCT ID	Phase	Start date	Sponsor(s)
NCT04179409	Phase 2	Jan 1, 2020	Ohio State University, Sarepta Therapeutics
NCT03992430	Phase 3	Aug 31, 2019	Sarepta Therapeutics
NCT04004065	Phase 2	Jun 26, 2019	Sarepta Therapeutics
NCT03985878	Phase 2	Jun 19, 2019	Sarepta Therapeutics
NCT03675126	Phase 1/Phase 2 (Phase 2)	Nov 15, 2018	Sarepta Therapeutics
NCT03375255	Phase 1	Dec 11, 2017	Sarepta Therapeutics
NCT03218995	Phase 2	Aug 16, 2017	Sarepta Therapeutics
NCT02420379	Phase 2	May 1, 2015	Sarepta Therapeutics
NCT02286947	Phase 2	Nov 1, 2014	Sarepta Therapeutics
NCT02255552	Phase 3	Sep 1, 2014	Sarepta Therapeutics
NCT01540409	Phase 2	Feb 1, 2012	Ohio State University, Sarepta Therapeutics
NCT01396239	Phase 2	Jul 1, 2011	Ohio State University, Sarepta Therapeutics
NCT00844597	Phase 1/Phase 2 (Phase 2)	Jan 1, 2009	Medical Research Council, Sarepta Therapeutics
NCT00159250	Phase 1/Phase 2 (Phase 2)	Oct 1, 2007	Department of Health, United Kingdom, Newcastle University, University of London

Showing 14 of 14 trials

Page 1 / 1

Organizations

Research & Development (6)

Organization	Org type	Trials	As lead sponsor	Phases	Earliest year
Sarepta Therapeutics	For profit	13	12	3	2009
Ohio State University	Academic/Hospital	3	1	1	2011
Department of Health, United Kingdom	Government	1	0	1	2007
Medical Research Council	Government	1	0	1	2009
Newcastle University	Academic/Hospital	1	0	1	2007
University of London	Academic/Hospital	1	1	1	2007

6 organizations

Page 1 / 1

Marketing (2)

Organization	Org type	Relationship	Date
Sarepta Therapeutics	For profit	NDA2	Sep 19, 2016
Sarepta Therapeutics	For profit	NDA	Sep 19, 2016