Eteplirsen

(Exondys 51®)

Eteplirsen

Drug updated on 10/30/2024

Dosage FormInjection (intravenous; 100 mg/2 mL, 500 mg/10 mL)
Drug ClassTransthyretin-directed antisense oligonucleotides
Ongoing and
Completed Studies
ClinicalTrials.gov

Indication

  • Indicated 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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Summary
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  • This summary is based on the review of five systematic review(s)/meta-analysis(es). [1-5]
  • Functional Outcomes: Eteplirsen significantly improved 6-minute walking distance (6MWD) with a mean difference of 67.3 meters at 48 weeks and 151.0 meters at 3 years. It also enhanced forced vital capacity (%pFVC) by 1.8% and maximal inspiratory pressure (%pMIP) by 4.4%. Ataluren and Drisapersen showed non-significant effects in individual trials, with meta-analysis indicating a significant 6MWD improvement of 18.3 meters for Ataluren and 21.5 meters for Drisapersen.
  • Prolonged Ambulation: Eteplirsen and Ataluren were associated with prolonged ambulation in patients, though no specific quantitative measures for ambulation duration were provided.
  • Comparative Effectiveness: Eteplirsen showed a more substantial impact on 6MWD, with improvements of 67.3 and 151 meters, compared to Ataluren (18.3 meters) and Drisapersen (21.5 meters).
  • There is no safety information available in the reviewed studies.
  • Clinically relevant population subgroups include children and adults with Duchenne muscular dystrophy (DMD) across various regions (Europe, Asia, North America), with specific exon deletions (e.g., exon 51) amenable to exon skipping associated with prolonged ambulation, as well as subgroups with milder phenotypes benefiting from exon 51 skipping treatments like Eteplirsen.

Product Monograph / Prescribing Information

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

Systematic Reviews / Meta-Analyses

Clinical Practice Guidelines

Document TitleYearSource
Update of the Brazilian consensus recommendations on Duchenne muscular dystrophy2023Arquivos de Neuro-Psiquiatria