FDA-Approved Exon‑Skipping Therapies for DMD & Key Trials/Programs

 

Drug NameBrand NameSponsorFDA Approval StatusPatient PopulationKey Trials / Programs
EteplirsenExondys 51Sarepta TherapeuticsAccelerated Approval (Sept 2016)DMD with mutations amenable to exon 51 skipping (~13%)PROMOVI (4658‑301); MISSION (4658‑402) — high‑dose level trial of eteplirsen
GolodirsenVyondys 53Sarepta TherapeuticsAccelerated Approval (Dec 2019)DMD with mutations amenable to exon 53 skipping (~8%)ESSENCE (4045‑301)
CasimersenAmondys 45Sarepta TherapeuticsAccelerated Approval (Feb 2021)DMD with mutations amenable to exon 45 skipping (~8%)ESSENCE (4045‑301)
ViltolarsenViltepsoNS PharmaAccelerated Approval (Aug 2020)DMD with mutations amenable to exon 53 skipping (~8%)Phase 2 + Long‑term Extension studies

Notes (Dated 1/6/2026):

  • All therapies above are under accelerated approval based on increases in dystrophin (surrogate endpoint); none have been converted to full (traditional) approval yet pending confirmatory evidence.
  • MISSION (MIS51ON; Study 4658‑402) is a high‑dose level program/trial of eteplirsen evaluating doses above the labeled 30 mg/kg to assess potential improvements in efficacy; it is not a separate drug.
  • Combined, these exon‑skipping therapies cover ~30% of the DMD population with amenable mutations (exons 45/51/53).

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