Drug updated on 9/4/2024
Dosage Form | Injection (intravenous; 250 mg/5 mL [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.
Latest News
Summary
- 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.
Product Monograph / Prescribing Information
Document Title | Year | Source |
---|---|---|
Viltepso (viltolarsen) Prescribing Information. | 2021 | NS Pharma, Inc., Paramus, NJ |
Randomized Controlled Trials
Document Title | Sex Distribution | Year | Source |
---|---|---|---|
Efficacy and safety of viltolarsen in boys with duchenne muscular dystrophy: Results from the phase 2, open-label, 4-year extension study. | 16Subjects F: null% M: 100% | 2023 | Journal of Neuromuscular Diseases |
Long-term functional efficacy and safety of viltolarsen in patients with duchenne muscular dystrophy. | 16Subjects F: null% M: 100% | 2022 | Journal of Neuromuscular Diseases |
Safety, tolerability, and efficacy of viltolarsen in boys with duchenne muscular dystrophy amenable to exon 53 skipping: a phase 2 randomized clinical trial. | 16Subjects F: 0% M: 100% | 2020 | JAMA Neurol |
Sex Distribution:
F:%
M:100%
16Subjects
Year:
2023
Source:Journal of Neuromuscular Diseases
Document Title
Sex Distribution:
F:%
M:100%
16Subjects
Year:
2022
Source:Journal of Neuromuscular Diseases
Sex Distribution:
F:0%
M:100%
16Subjects
Year:
2020
Source:JAMA Neurol
Clinical Practice Guidelines
Document Title | Year | Source |
---|---|---|
Evolving role of viltolarsen for treatment of duchenne muscular dystrophy. | 2024 | Advances in Therapy |