Wednesday, July 8, 2026

Drug profile · INN

viltolarsen

viltolarsen (VILTEPSO) is a dystrophin pre-mrna positive modulator. Route of administration: intravenous. Current US regulatory status: approved.

FDA: approved PMDA: approved
US status approved
Patents linked 0
Pipeline programs 1
News articles 0
Drug details — viltolarsen
US status
approved
Class
Route
INTRAVENOUS
Patents
0
Programs
1
Data quality
0.84

Quick answer

viltolarsen (VILTEPSO) is a dystrophin pre-mrna positive modulator. Route of administration: intravenous. Current US regulatory status: approved.

Key facts

INN viltolarsen
Brand VILTEPSO
Mechanism Dystrophin pre-mRNA positive modulator
Target Dystrophin pre-mRNA
Route INTRAVENOUS
Formulation SOLUTION

Mechanism of action

Dystrophin pre-mRNA positive modulator

Primary target: Dystrophin pre-mRNA

Approval history

Authority Status Date
FDA approved
PMDA approved

Pipeline programs

  1. Viltolarsen Phase 3 · Anaxis Pharma · Duchenne Muscular Dystrophy (DMD)

Related & competitor drugs

  1. casimersen Dystrophin pre-mRNA positive modulator
  2. eteplirsen Musculo-skeletal system (M09)
  3. golodirsen Dystrophin pre-mRNA positive modulator

Frequently asked questions

What is viltolarsen?

viltolarsen (VILTEPSO) is a dystrophin pre-mrna positive modulator. Route of administration: intravenous. Current US regulatory status: approved.

What is the brand name for viltolarsen?

VILTEPSO is a marketed brand name for viltolarsen. Check FDA Drugs@FDA for the current US label holder and prescribing information.

What is the US approval status of viltolarsen?

viltolarsen has approved status in the United States according to our Drugs@FDA-derived records. Verify on the official FDA label before clinical or commercial decisions.