Drug updated on 4/18/2024
| Dosage Form | Injection (intravenous; 50 mg/mL) |
| Drug Class | 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 53 skipping.
Summary
- Golodirsen (Vyondys 53) is 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 53 skipping.
- A single systematic review/meta-analysis focused on its safety, efficacy, and comparison with other treatments for DMD, specifically in patients amenable to exon 53 skipping.
- The drug demonstrated a favorable safety profile during the study. Most adverse events were classified as mild, non-serious, and unrelated to golodirsen. There were no reported cases of safety-related discontinuations or deaths among participants.
- In terms of efficacy, golodirsen showed significant biologic activity with an increase in dystrophin protein expression and exon skipping. Patients treated with this drug experienced a lesser decline in their functional outcomes compared to external controls; however, statistical significance was not always reached.
- No direct comparisons between golodirsen and other DMD treatments were provided within this study, but it appears more effective for those carrying specific genetic mutations targeted by this drug due to its precision medicine approach.
- While there are limitations regarding definitive ranking against other treatments due to the lack of direct comparisons within the study reviewed, data suggest that Vyondys 53 represents a promising option given its improvement on biological markers related to disease progression along with a notable long-term safety profile.
Product Monograph / Prescribing Information
| Document Title | Year | Source |
|---|---|---|
| Vyondys 53 (golodirsen) Prescribing Information. | 2021 | Sarepta Therapeutics, Inc., Cambridge, MA |