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Exondys 51 (ETEPLIRSEN)
Exondys 51 binds to specific RNA sequences to prevent the skipping of exon 51 during dystrophin mRNA splicing.
Exondys 51 (eteplirsen) is an antisense oligonucleotide developed by Sarepta Therapeutics Inc, targeting exon 51 of dystrophin pre-mRNA to treat Duchenne muscular dystrophy. It was FDA-approved in 2016 and remains a patented product. Exondys 51 works by binding to specific RNA sequences, preventing the skipping of exon 51 during dystrophin mRNA splicing, which leads to the production of a partially functional dystrophin protein. The treatment is designed to slow disease progression in patients with Duchenne muscular dystrophy. Key safety considerations include potential liver enzyme elevations and injection site reactions.
At a glance
| Generic name | ETEPLIRSEN |
|---|---|
| Sponsor | Sarepta |
| Drug class | Antisense Oligonucleotide [EPC] |
| Target | exon 51 of dystrophin pre-mRNA |
| Modality | Oligonucleotide |
| Therapeutic area | Rare Disease |
| Phase | FDA-approved |
| First approval | 2016 |
| Annual revenue | 300 |
Mechanism of action
Eteplirsen is designed to bind to exon 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 and Study [see Clinical Studies (14)].
Approved indications
- Duchenne muscular dystrophy
Common side effects
- Balance disorder
- Vomiting
- Contact dermatitis
- Headache
- Cough
- Rash
- Hypersensitivity reactions
- Bronchospasm
- Cyanosis of the lips
- Malaise
- Pyrexia
- Flushing
Key clinical trials
- A Study to Compare Safety and Efficacy of High Doses of Eteplirsen in Participants With Duchenne Muscular Dystrophy (DMD) (MIS51ON) (PHASE3)
- A Long-term Observational Study Evaluating Eteplirsen, Golodirsen, or Casimersen in Routine Clinical Practice
- 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. (PHASE2)
- A Study to Evaluate Safety, Tolerability, and Efficacy of Eteplirsen in Participants With Duchenne Muscular Dystrophy (DMD) Who Have Completed Study 4658-102 (NCT03218995) (PHASE2)
- Study of Eteplirsen in Young Participants With Duchenne Muscular Dystrophy (DMD) Amenable to Exon 51 Skipping (PHASE2)
- Safety Study of Eteplirsen to Treat Early Stage Duchenne Muscular Dystrophy (PHASE2)
- Study of Eteplirsen in DMD Patients (PHASE3)
- Efficacy, Safety, and Tolerability Rollover Study of Eteplirsen in Subjects With Duchenne Muscular Dystrophy (PHASE2)
Patents
| Patent | Expiry | Type |
|---|---|---|
Primary sources
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| FDA label | Mechanism, indications, dosing, boxed warnings, drug interactions |
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |
| FDA Orange Book | Patents + exclusivity |
| SEC EDGAR | Revenue + earnings |
Competitive intelligence
For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:
- Exondys 51 CI brief — competitive landscape report
- Exondys 51 updates RSS · CI watch RSS
- Sarepta portfolio CI