(Exondys 51®)

Exondys 51®

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Drug updated on 4/26/2024

Dosage FormInjection (intravenous; 100 mg/2 mL, 500 mg/10 mL)
Drug ClassAntisense oligonucleotides
Ongoing and
Completed Studies


  • For the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 51 skipping.

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  • Eteplirsen (Exondys 51) is indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients with a confirmed mutation of the DMD gene that is amenable to exon 51 skipping. Its mechanism promotes exon 51 skipping, enabling the production of partially functional dystrophin crucial for muscle function.
  • Three systematic reviews/meta-analyses focused on treating and managing Duchenne muscular dystrophy compared the safety and effectiveness of Exondys 51 with other treatments like ataluren and drisapersen.
  • In terms of effectiveness, eteplirsen demonstrated significant improvement in the six-minute walk distance (+67.3 m at 48 weeks and +151.0 m at three years), indicating its potential in slowing disease progression better than ataluren or drisapersen, which only improved by +18.3m and +21.5m, respectively.
  • Improvements were also noted in forced vital capacity (∆%pFVC = +1.8%) and maximal inspiratory pressure (∆%pMIP = +4.4%), suggesting a beneficial impact on respiratory function from using eteplirsen as opposed to other treatments under review.
  • The efficacy of these treatments depends on specific mutations within the DMD gene being present that allow for exon-skipping or read-through mechanisms, such as those targeted by eteplirsen, golodirsen, or ataluren therapies.
  • Despite promising outcomes associated with these treatments, including Exondys 51 (eteplirsen), calls for more clinical trials to confirm efficacy alongside quality-of-life studies were noted, due to incomplete understanding about long-term benefits impacting life quality from the current research data available.

Product Monograph / Prescribing Information

Document TitleYearSource
Exondys 51 (eteplirsen) Prescribing Information.2022Sarepta Therapeutics, Inc., Cambridge, MA

Systematic Reviews / Meta-Analyses

Clinical Practice Guidelines