Exondys 51
(Eteplirsen)Phase 3RNA therapy
Sarepta TherapeuticsExondys 51 is a rna therapy that works as a Exon-Skipping Antisense Oligonucleotide.[2] It is being investigated across 1 indication, with its most advanced program in Phase 3.[1]
Exondys 51 is one of 24 aso programs targeting Duchenne Muscular Dystrophy. It is currently in Phase 3.
1
Indications
7
Total Trials
2
Active Trials
23
Competitors
10
FDA Applications
NDA/BLA/ANDA regulatory filings
5
Patents
Pipeline
Status by Indication[3][1]
Indication
Competition
Competitive Landscape
40 competitors in Duchenne Muscular Dystrophy
#DrugCompanyPhaseTrials
1Isordil
AstraZenecaApproved
Deltasone
PPfizer's Upjohn has merged with Mylan to form Viatris Inc.
Approved
Coreg
Akero TherapeuticsApproved
Rapamune
PPrism Pharmaceuticals
Approved
Garamycin
SSchering-Plough
Approved
Advil
KKenvue Brands
Approved
Rifadin
SanofiPhase 3
Duvyzat
ItalfarmacoPhase 3
Vyondys 53
Sarepta TherapeuticsApproved
Agamree
CCatalyst Pharmaceuticals, Inc.
Approved
Ranked by pipeline maturity and clinical activity. Current drug highlighted in teal.
Timeline
Expected Milestones
Clinical
Trials by Indication[1]
Duchenne Muscular Dystrophy5 trials
Observational Study1 trial
Regulatory
FDA Approval Timeline[4]
Sep 19, 2016
EXONDYS 51
NDA NDA206488
Sep 19, 2016
EXONDYS 51
NDA NDA206488
Feb 22, 2017
EXONDYS 51
NDA NDA206488
Feb 8, 2018
EXONDYS 51
NDA NDA206488
Oct 11, 2018
EXONDYS 51
NDA NDA206488
Jul 7, 2020
EXONDYS 51
NDA NDA206488
Jan 18, 2022
EXONDYS 51
NDA NDA206488
Jan 18, 2022
EXONDYS 51
NDA NDA206488
Jan 18, 2022
EXONDYS 51
NDA NDA206488
Dec 27, 2024
EXONDYS 51
NDA NDA206488
Sources
References
- [1]
- [2]
- [3]Open Targets Platform — Drug–disease associations for Eteplirsen. EMBL-EBI & Wellcome Sanger Institute.
- [4]
- [5]
- [6]
- [7]