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Exondys 51
At a glance
| Generic name | Exondys 51 |
|---|---|
| Also known as | Eteplirsen, AVI-4658 |
| Sponsor | Kevin Flanigan |
| Modality | Small molecule |
| Phase | FDA-approved |
Approved indications
Common side effects
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)
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 |
|---|---|
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |
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
- Kevin Flanigan portfolio CI