Summary

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• Vyondys 53 (golodirsen) 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 53 skipping.
• This summary is based on the review of two systematic review(s)/meta-analysis(es). ^[1-2]
• Dystrophin Protein Expression: Golodirsen treatment led to a 16.0-fold increase in dystrophin protein expression (P < 0.001), with a mean percent normal dystrophin protein standard of 1.019% at week 48 (range 0.09%–4.30%). 4. 6-Minute Walk Test (6MWT): After 3 years, golodirsen-treated patients showed a change from baseline in the 6MWT of -99.0 meters compared to -181.4 meters in external controls (P = 0.067), with loss of ambulation in 9% of golodirsen-treated patients versus 26% in external controls (P = 0.21).
• Forced Vital Capacity (FVC%p): Golodirsen-treated patients experienced an 8.4% decline in FVC%p over 3 years, which was favorable compared to literature-reported rates.
• Adverse events reported were generally mild, nonserious, and unrelated to golodirsen, with no safety-related discontinuations or deaths observed.
• Safety findings aligned with prior observations in pediatric DMD patients, with the majority of the study drug excreted within 4 hours post-administration.
• There is no population types or subgroups information available in the reviewed studies.