Drug updated on 4/26/2024
| Dosage Form | Injection (intravenous; 100 mg/2 mL, 500 mg/10 mL) |
| Drug Class | Antisense oligonucleotides |
| Ongoing and Completed Studies | ClinicalTrials.gov |
Indication
- 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.
Summary
- 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 Title | Year | Source |
|---|---|---|
| Exondys 51 (eteplirsen) Prescribing Information. | 2022 | Sarepta Therapeutics, Inc., Cambridge, MA |
Systematic Reviews / Meta-Analyses
| Document Title | Year | Source |
|---|---|---|
| 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 Practice Guidelines
| Document Title | Year | Source |
|---|---|---|
| Clinical practice guidelines for the diagnosis and management of Duchenne muscular dystrophy: A scoping review. | 2023 | Frontiers in Neurology |