Summary

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• Viltepso (viltolarsen) 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.
• This summary is based on the review of three randomized controlled trial(s). ^[1-3]
• Primary Efficacy Outcome (Time to Stand from Supine - TTSTAND): Viltolarsen-treated patients exhibited stabilization of motor function over two years and significant slowing of disease progression compared to the CINRG DNHS control group, which showed a decline. The improvement in TTSTAND was significant when compared to age-matched and treatment-matched natural history controls.
• Secondary Efficacy Outcomes: Viltolarsen-treated participants demonstrated stabilization in the time to run/walk 10 meters from baseline through week 109, with significant improvement observed at the week 25 visit compared to controls. The 6-minute walk test also showed significant improvement at the week 25 visit compared to controls.
• Dystrophin Production: Significant drug-induced dystrophin production was reported in both viltolarsen dose cohorts (40 mg/kg per week and 80 mg/kg per week).
• Viltolarsen was well tolerated, with most treatment-emergent adverse events reported as mild or moderate in severity. No treatment-emergent adverse events required dose reduction, interruption, or discontinuation of the study drug. There were no serious adverse events or deaths during the study.
• Safety was assessed continuously over the long-term extension study, with no participants discontinuing the drug due to adverse events, and no specific adverse events were detailed beyond their general categorization as mild or moderate.
• There is no population types or subgroups information available in the reviewed studies.