Substance / Medication

Viltolarsen

Overview

Active Ingredient
viltolarsen
RxNorm CUI
2389840

Indications

14 [see Clinical Studies ()]. VILTEPSO 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 indication is approved under accelerated approval based on an increase in dystrophin production in skeletal muscle observed in patients treated with VILTEPSOContinued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial.

Labeler: NS Pharma, Inc.Updated: 2025-07-16T00:00:00.000ZFull label on DailyMed

Contraindications

When this intervention should not be used

None.

Indications & Related Conditions

Conditions associated via SNOMED clinical relationships

Administration & Protocol

Dosing, route, and treatment protocol

Detailed dosage and administration information is available in the full FDA drug label.

View full prescribing information on DailyMed

Monitoring & Follow-Up

Biomarkers relevant to this intervention via related conditions

No monitoring biomarkers have been mapped yet. Biomarker-intervention linkages are derived through related conditions and will expand as the knowledge graph grows.

Clinical Trials

1 trials linked to this intervention

1
Total Trials
1
Recruiting
0
With Results

Research Evidence

Published studies and systematic reviews

Sort:
Evolving Role of Viltolarsen for Treatment of Duchenne Muscular Dystrophy.
Vincik LeighAnn Y, Dautel Alexandra D, Staples Abigail A et al. · Adv Ther · 2024
PMID: 38376743Review
Patient With Duchenne Muscular Dystrophy Who Tolerated Viltolarsen After Prior Anaphylaxis to Golodirsen.
Bassell-Hawkins Julia, Piccoli Cara, Rocha Carolina Tesi · Muscle Nerve · 2025
PMID: 40726380Other
Safety and efficacy of viltolarsen in ambulatory and nonambulatory males with Duchenne muscular dystrophy.
Harper Amy D, Topaloglu Haluk, Mercuri Eugenio et al. · Sci Rep · 2024
PMID: 39379403TrialFull text (PMC)
Viltolarsen: From Preclinical Studies to FDA Approval.
Roshmi Rohini Roy, Yokota Toshifumi · Methods Mol Biol · 2023
PMID: 36401022Preclinical
Prediction of Human Pharmacokinetics of Phosphorodiamidate Morpholino Oligonucleotides in Duchenne Muscular Dystrophy Patients Using Viltolarsen.
Imai Shunji, Suda Yusuke, Mori Jumpei et al. · Drug Metab Dispos · 2023
PMID: 37468285Preclinical
Long-Term Functional Efficacy and Safety of Viltolarsen in Patients with Duchenne Muscular Dystrophy.
Clemens Paula R, Rao Vamshi K, Connolly Anne M et al. · J Neuromuscul Dis · 2022
PMID: 35634851TrialFull text (PMC)

Research data from MEDLINE/PubMed

Benefits & Expected Outcomes

Benefits, expected outcomes, efficacy data, and NNT (Number Needed to Treat) are pending physician authorship and evidence review.

Risks & Side Effects

Adverse reaction and safety data for this drug is sourced from the FDA-approved label.

View adverse reactions & drug interactions on DailyMed

Related Symptoms

Symptoms associated with conditions this intervention addresses

No related symptoms have been mapped yet. Symptom linkages are derived through associated conditions.

Alternatives & Comparisons

Alternative treatments, comparison data, and clinical decision support are pending physician authorship.

Medical Disclaimer

This information is for educational purposes only and does not constitute medical advice, diagnosis, or treatment. Always consult with a qualified healthcare provider regarding any medical condition or treatment plan.

Do not start, stop, or change any treatment without consulting your healthcare provider.

Quick Facts

Type
Substance / Medication
Fully Specified Name
Viltolarsen (substance)
SNOMED CT
897124003
UMLS CUI
C5139804
RxNorm CUI
2389840
Labeler
NS Pharma, Inc.

Clinical Data

This intervention maps to 2 entities in the Healos knowledge graph.

2
Conditions
0
Biomarkers
0
Specialists
0
Symptoms
1
Clinical Trials

Data is sourced from SNOMED CT, UMLS, and the Healos clinical knowledge graph. Content sections marked as pending require physician authorship. Consult a healthcare provider before starting any treatment.