VILTEPSO (viltolarsen)
VILTEPSO is indicated for the treatment of Duchenne Muscular Dystrophy.
How VILTEPSO Works
Viltolarsen is an antisense oligonucleotide that binds to exon 53 of the dystrophin pre-mRNA. This binding triggers the exclusion (skipping) of this exon during mRNA processing. By restoring the reading frame, the drug allows for the production of an internally truncated but functional dystrophin protein in patients with amenable mutations.
Development Insights
Details
- Status
- Prescription
- First Approved
- 2020-08-12
- Patent Cliff
- 2034
- Routes
- INTRAVENOUS
- Dosage Forms
- SOLUTION
VILTEPSO Approval History
What VILTEPSO Treats
1 indicationsVILTEPSO is approved for 1 conditions since its original approval in 2020. These indications span multiple therapeutic areas including oncology, immunology, and more.
- Duchenne Muscular Dystrophy
VILTEPSO Competitive Set
ProThree rings of competition based on shared molecular targets and treated indications.
Direct competitors
Same target(s) AND same indication — head-to-head.
Indication competitors
Same indication, different mechanism — what else might this patient receive?
Filters applied: drops same-active-ingredient (505(b)(2) reformulations), route-mismatch (topical vs systemic), and cross-therapeutic-area matches in same-indication rings.
What's emerging in VILTEPSO's indications
See all emerging drugs →Phase 3 candidates targeting molecules with no FDA-approved drug, in indications VILTEPSO treats. First-in-class if their pivotal trials read out positive.
Drugs Similar to VILTEPSO
3 of 9FDA-approved drugs for similar conditions. Compare mechanisms and indications to understand treatment alternatives.
Clinical Trial Registry
3 trials| Trial | Sponsor ID | Phase | Status | Title |
|---|---|---|---|---|
| NCT04060199 results posted | NS-065/NCNP-01-301 | Ph 3 | completed | Study to Assess the Efficacy and Safety of Viltolarsen in Ambulant Boys With DMD (RACER53) |
| NCT04956289 results posted | NS-065/NCNP-01-211 | Ph 2 | completed | Study to Assess the Safety, Tolerability, and Efficacy of Viltolarsen in Ambulant and Non-Ambulant Boys With DMD (Galactic53) |
| NCT04687020 | VILT-502 | Ph 4 | active not recruiting | Long-term Use of Viltolarsen in Boys With Duchenne Muscular Dystrophy in Clinical Practice (VILT-502) |
Active Pipeline
Ongoing clinical trials by development phase
Key Completed Trials
Completed studies with published results, ranked by significance
Trial Timeline
Full development history with FDA approval milestones
Understanding FDA Approval Types
| Count | Type | What it means |
|---|---|---|
| - | ORIG | Original approval - drug first enters market |
| - | SUPPL - Efficacy | New indication (new disease/condition approved) |
| - | SUPPL - Labeling | Label text changes (warnings, dosing updates) |
| - | SUPPL - Manufacturing | Production changes (new facility) |
| - | SUPPL - Chemistry | Formulation changes (new dosage strength) |
Green lines in the timeline show ORIG and Efficacy approvals - the clinically meaningful milestones.
VILTEPSO FDA Label Details
Indications & Usage
FDA Label (PDF)VILTEPSO is indicated for the treatment of Duchenne Muscular Dystrophy.
VILTEPSO Patents & Exclusivity
Patents (2 active)
Exclusivity
Pro Intelligence Preview
Deep insights for VILTEPSO
Revenue Insights
- • Quarterly revenue tracking
- • Historical trend analysis
Patent Timeline
- • Cliff: 2034
- • 2 active patents
Trial Analysis
- • 3 total trials
- • Stage: Expansion
Competitive Landscape
- • 9 similar drugs
- • Same target/indication analysis
Full approval history • All patents • Revenue trends • Competitor analysis
Data Sources
Data sourced from official FDA and NIH databases. Click links to verify on original sources.
How We Calculate These Metrics
Trial Activity Stage
Measures the current development activity pattern based on trial phases, status, and trends. Important: This measures R&D activity, not commercial lifecycle.
Trial statuses: "Active" means recruiting or ongoing. "Completed" means reached planned endpoint. "Terminated" means stopped early—often due to safety, efficacy, or business reasons.
- Growth: High proportion of early-phase trials (Phase 1/2), active development
- Expansion: Significant Phase 3 activity, approaching or pursuing approvals
- Mature: Substantial Phase 4 post-marketing studies
- Stable: Mixed phase distribution, steady development
- Declining: Low active trial ratio, reduced R&D investment