NDAINTRAVENOUSSOLUTIONPriority Review
Approved
Sep 2016
Lifecycle
Peak
Competitive Pressure
0/100
Clinical Trials
12
Data completeness
5/8 — Partial
Priority Review
EXONDYS 51 (eteplirsen) by Sarepta Therapeutics is 51 of dystrophin pre-mrna, resulting in exclusion of this exon during mrna processing in patients with genetic mutations that are amenable to exon 51 skipping. First approved in 2016.
Drug data last refreshed 8h ago · AI intelligence enriched 3d ago
AI summarySynthesized from FDA + DailyMed + ClinicalTrials.gov data
EXONDYS 51 (eteplirsen) is an antisense oligonucleotide that promotes exon 51 skipping in dystrophin pre-mRNA, allowing production of internally truncated functional dystrophin protein in eligible patients. It is administered intravenously and targets patients with Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping. This precision mechanism represents a landmark shift toward genetic tailoring in rare, severe neuromuscular disease.
Peak7.5 years to LOE
Peak-stage orphan therapy with established payer infrastructure; commercial teams are focused on patient identification, access optimization, and market penetration in a small but high-value patient population.
Mechanism of Action
51 of dystrophin pre-mRNA, resulting in exclusion of this exon during mRNA processing in patients with genetic mutations that are amenable to exon 51 skipping. Exon skipping is intended to allow for production of an internally truncated dystrophin protein, which was evaluated in Study 2 and Study 3…
Pharmacologic Class:
Antisense Oligonucleotide
Indications
Indication data is being enriched from DailyMed and FDA labeling. Check back soon for approved therapeutic uses.
Clinical Trials (12)
A Study to Compare Safety and Efficacy of High Doses of Eteplirsen in Participants With Duchenne Muscular Dystrophy (DMD) (MIS51ON)
Started Jul 2020
160 enrolled
Muscular Dystrophy, Duchenne
A 48-Week, Open Label, Study to Evaluate the Efficacy and Safety of AMONDYS 45, EXONDYS 51, VYONDYS 53 in Subjects With DuchenneMuscular Dystrophy Carrying Eligible DMD Duplications.
Started Feb 2020
3 enrolled
Duchenne Muscular Dystrophy
A Study to Evaluate Safety, Tolerability, and Efficacy of Eteplirsen in Participants With Duchenne Muscular Dystrophy (DMD) Who Have Completed Study 4658-102 (NCT03218995)
Started Jun 2019
15 enrolled
Duchenne Muscular Dystrophy
Two-Part Study for Dose Determination of Vesleteplirsen (SRP-5051) (Part A), Then Dose Expansion (Part B) in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment
Started Jun 2019
62 enrolled
Duchenne Muscular Dystrophy
A Long-term Observational Study Evaluating Eteplirsen, Golodirsen, or Casimersen in Routine Clinical Practice
Started Jan 2019
300 enrolled
Duchenne Muscular Dystrophy
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Data Sources
- Drug label: DailyMed / FDA
- Clinical trials: ClinicalTrials.gov
- Patent data: FDA Orange Book
- Spending data: CMS Medicare
The information on this page is for informational purposes only and should not be used as a substitute for professional medical advice. Drug information is sourced from FDA, DailyMed, and other government databases. Adverse event data from FAERS does not establish causation. Always consult a healthcare professional for medical decisions.