Drug updated on 12/11/2024
| Dosage Form | Injection (intravenous; 100 mg/2 mL, 500 mg/10 mL) |
| Drug Class | Transthyretin-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.
Latest News
Summary
- 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 Title | Year | Source |
|---|---|---|
| Exondys 51 (eteplirsen) Prescribing Information. | 2022 | Sarepta Therapeutics, Inc., Cambridge, MA |
Systematic Reviews / Meta-Analyses
| Document Title | Year | Source |
|---|---|---|
| Predictors of Loss of Ambulation in Duchenne Muscular Dystrophy: A Systematic Review and Meta-Analysis | 2024 | Journal of Neuromuscular Diseases |
| Factors Associated with Respiratory Health and Function in Duchenne Muscular Dystrophy: A Systematic Review and Evidence Grading | 2024 | Journal of Neuromuscular Diseases |
| Restorative treatments of dystrophin expression in Duchenne muscular dystrophy: A systematic review | 2020 | Annals of Clinical and Translational Neurology |
| Muscle and cardiac therapeutic strategies for Duchenne muscular dystrophy: past, present, and future | 2020 | Pharmacological Reports |
| Clinical Phenotypes of DMD Exon 51 Skip Equivalent Deletions: A Systematic Review | 2020 | Journal of Neuromuscular Diseases |
Clinical Practice Guidelines
| Document Title | Year | Source |
|---|---|---|
| Update of the Brazilian consensus recommendations on Duchenne muscular dystrophy | 2023 | Arquivos de Neuro-Psiquiatria |