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 41 aso programs targeting Duchenne Muscular Dystrophy. It is currently in Phase 3.
1
Indications
7
Total Trials
2
Active Trials
40
Competitors
1
FDA Application
NDA/BLA/ANDA regulatory filings
5
Patents
Pipeline
Status by Indication[3][1]
Indication
Competition
Competitive Landscape
125 competitors in Duchenne Muscular Dystrophy
#DrugCompanyPhaseTrials
1Cialis
Eli LillyPhase 3
Aldactone
PfizerPhase 3
Rifadin
SanofiPhase 3
Nolvadex
AstraZenecaPhase 3
Avicel
SSanthera Pharmaceuticals
Phase 3
Rayos
RRoxane Laboratories
Phase 3
Inspra
PPfizer's Upjohn has merged with Mylan to form Viatris Inc.
Phase 3
Duvyzat
ItalfarmacoPhase 3
Bystolic
VVitae Pharmaceuticals Inc., an Allergan affiliate
Phase 3
ENSPRYNG
RochePhase 2
Ranked by pipeline maturity and clinical activity. Current drug highlighted in teal.
Timeline
Expected Milestones
Clinical
Trials by Indication[1]
Duchenne Muscular Dystrophy5 trials
Regulatory
FDA Approval Timeline[4]
Sep 19, 2016
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]