Last reviewed · How we verify
Viltepso (VILTOLARSEN)
Viltepso works by binding to exon 53 of dystrophin pre-mRNA, allowing for the production of a partially functional dystrophin protein.
At a glance
| Generic name | VILTOLARSEN |
|---|---|
| Sponsor | Nippon Shinyaku |
| Target | exon 53 of dystrophin pre-mRNA |
| Modality | Oligonucleotide |
| Therapeutic area | Rare Disease |
| Phase | FDA-approved |
| First approval | 2020 |
Mechanism of action
VILTEPSO is designed to bind to exon 53 of dystrophin pre-mRNA resulting in exclusion of this exon during mRNA processing in patients with genetic mutations that are amenable to exon 53 skipping. Exon 53 skipping is intended to allow for production of an internally truncated dystrophin protein in patients with genetic mutations that are amenable to exon 53 skipping.
Approved indications
- Duchenne muscular dystrophy
Common side effects
- Upper respiratory tract infection
- Injection site reaction
- Cough
- Pyrexia
- Contusion
- Arthralgia
- Diarrhea
- Vomiting
- Abdominal pain
- Ejection fraction decreased
- Urticaria
- Anti-dystrophin antibodies
Key clinical trials
- Safety and Dose Finding Study of NS-065/NCNP-01 in Boys With Duchenne Muscular Dystrophy (DMD) (PHASE2)
- Study to Assess the Efficacy and Safety of Viltolarsen in Ambulant Boys With DMD (RACER53) (PHASE3)
- Study to Assess the Safety, Tolerability, and Efficacy of Viltolarsen in Ambulant and Non-Ambulant Boys With DMD (Galactic53) (PHASE2)
- Study to Assess the Safety and Efficacy of Viltolarsen in Ambulant Boys With DMD (RACER53-X) (PHASE3)
- Long-term Use of Viltolarsen in Boys With Duchenne Muscular Dystrophy in Clinical Practice (VILT-502) (PHASE4)
- The Expanded Access Use of Viltolarsen in Duchenne Muscular Dystrophy With Confirmed Exon 53 Amenable Mutation
Patents
| Patent | Expiry | Type |
|---|---|---|
Primary sources
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| FDA label | Mechanism, indications, dosing, boxed warnings, drug interactions |
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |
| FDA Orange Book | Patents + exclusivity |
Competitive intelligence
For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:
- Viltepso CI brief — competitive landscape report
- Viltepso updates RSS · CI watch RSS
- Nippon Shinyaku portfolio CI