Viltepso

(Viltolarsen)

Phase 3RNA therapy

Viltepso is a rna therapy that works as a Exon-Skipping Antisense Oligonucleotide.[2] It is being investigated across 1 indication, with its most advanced program in Phase 3.[1]

Viltepso is one of 41 aso programs targeting Duchenne Muscular Dystrophy. It is currently in Phase 3.

Indications

Total Trials

Active Trials

Competitors

FDA Application

NDA/BLA/ANDA regulatory filings

Patents

Pipeline

Status by Indication[3][1]

Indication

Duchenne Muscular DystrophyActive, not recruiting

Duchenne Muscular DystrophyPhase 3

Active, not recruiting

Competition

Competitive Landscape

125 competitors in Duchenne Muscular Dystrophy

#DrugCompanyPhaseTrials

Cialis

Eli Lilly

Phase 3

2 2

Aldactone

Pfizer

Phase 3

1 3

Rifadin

Sanofi

Phase 3

1 4

Nolvadex

AstraZeneca

Phase 3

1 5

Avicel

SSanthera Pharmaceuticals

Phase 3

6 6

Rayos

RRoxane Laboratories

Phase 3

5 7

Inspra

PPfizer's Upjohn has merged with Mylan to form Viatris Inc.

Phase 3

2 8

Duvyzat

Italfarmaco

Phase 3

2 9

Bystolic

VVitae Pharmaceuticals Inc., an Allergan affiliate

Phase 3

1 10

ENSPRYNG

Roche

Phase 2

Ranked by pipeline maturity and clinical activity. Current drug highlighted in teal.

Timeline

Expected Milestones

Q1 2027

Phase 3Recruiting

Duchenne Muscular Dystrophy

NCT04060199

Q4 2032

Post-ApprovalActive, not recruiting

Duchenne Muscular Dystrophy

NCT04687020

Clinical

Trials by Indication[1]

Duchenne Muscular Dystrophy3 trials

The Expanded Access Use of Viltolarsen in Duchenne Muscular Dystrophy With Confirmed Exon 53 Amenable Mutation

Approved for marketing· NS Pharma logo

NS Pharma··NCT04337112

Long-term Use of Viltolarsen in Boys With Duchenne Muscular Dystrophy in Clinical Practice (VILT-502)

Active, not recruiting· NS Pharma logo

NS Pharma·Phase 4·NCT04687020·9 enrolled

RACER53: Viltolarsen in Duchenne Muscular Dystrophy

Recruiting· NS Pharma logo

NS Pharma·Phase 3·NCT04060199·74 enrolled

Safety and Efficacy1 trial

Study to Assess the Safety and Efficacy of Viltolarsen in Ambulant Boys With DMD (RACER53-X)

Unknown status· NS Pharma logo

NS Pharma·Phase 3·NCT04768062·74 enrolled

Efficacy1 trial

Study to Assess the Safety, Tolerability, and Efficacy of Viltolarsen in Ambulant and Non-Ambulant Boys With DMD (Galactic53)

Completed· NS Pharma logo

NS Pharma·Phase 2·NCT04956289·20 enrolled

Regulatory

FDA Approval Timeline[4]

Aug 12, 2020

VILTEPSO

NDA NDA212154

Sources

References

[1]
ClinicalTrials.gov — 5 trial records for Viltolarsen. U.S. National Library of Medicine, National Institutes of Health.
[2]
ChEMBL — Viltolarsen compound record. European Bioinformatics Institute (EMBL-EBI).
[3]
Open Targets Platform — Drug–disease associations for Viltolarsen. EMBL-EBI & Wellcome Sanger Institute.
[4]
OpenFDA — FDA approval records (NDA NDA212154). U.S. Food & Drug Administration.
[5]
FDA Orange Book — Patent and exclusivity data for Viltepso. U.S. Food & Drug Administration.
[6]
SEC EDGAR — Nippon Shinyaku Co., Ltd. company filings. U.S. Securities and Exchange Commission.

Details

Drug Info

Quick Facts

Mechanism of Action: Exon-Skipping Antisense Oligonucleotide[2]
Modality: RNA therapy[2]
Highest Phase: Phase 3[1]
Company: NNippon Shinyaku Co., Ltd.

Patent Landscape[5]

Patent Cliff

Aug 31, 2031

Earliest patent expiration — generic entry possible after this date

Patent #Expires

10870676Aug 31, 2031

9079934Apr 3, 2034

Viltepso

Status by Indication[3][1]

Competitive Landscape

Expected Milestones

Q1 2027

Q4 2032

Trials by Indication[1]

Duchenne Muscular Dystrophy3 trials

Safety and Efficacy1 trial

Efficacy1 trial

FDA Approval Timeline[4]

References

Drug Info

Quick Facts

Patent Landscape[5]

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