Vyondys 53

(Golodirsen)

ApprovedRNA therapy

Vyondys 53 is a rna therapy that works as a Exon 53 Skipping ASO.[2] It is being used across 1 indication, with its most advanced program in Approved.[1]

Vyondys 53 is one of 24 aso programs targeting Duchenne Muscular Dystrophy. It is currently in Approved.

Indications

Total Trials

Active Trials

Competitors

FDA Applications

NDA/BLA/ANDA regulatory filings

Patents

Pipeline

Status by Indication[3][1]

Indication

Duchenne Muscular DystrophyTerminated

Duchenne Muscular DystrophyApproved

Terminated

Competition

Competitive Landscape

40 competitors in Duchenne Muscular Dystrophy

#DrugCompanyPhaseTrials

Isordil

AstraZeneca

Approved

—2

Deltasone

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

Approved

2 3

Coreg

Akero Therapeutics

Approved

1 4

Rapamune

PPrism Pharmaceuticals

Approved

1 5

Garamycin

Advil

Rifadin

1 8

Duvyzat

Italfarmaco

Phase 3

2 9

Agamree

CCatalyst Pharmaceuticals, Inc.

Approved

5 10

Vyondys 53

Sarepta Therapeutics

Approved

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

Clinical

Trials by Indication[1]

Duchenne Muscular Dystrophy2 trials

An Open-Label Study of Golodirsen in Non-Ambulant Patients With Duchenne Muscular Dystrophy

Terminated·RRare Disease Research, LLC·Phase 4·NCT04708314·2 enrolled

An Extension Study to Evaluate Casimersen or Golodirsen in Patients With Duchenne Muscular Dystrophy

Terminated· Sarepta Therapeutics logo

Sarepta Therapeutics·Phase 3·NCT03532542·171 enrolled

Observational Study1 trial

A Long-term Observational Study Evaluating Eteplirsen, Golodirsen, or Casimersen in Routine Clinical Practice

Enrolling by invitation· Sarepta Therapeutics logo

Sarepta Therapeutics··NCT06606340·300 enrolled

Regulatory

FDA Approval Timeline[4]

Dec 12, 2019

VYONDYS 53

NDA NDA211970

Dec 12, 2019

VYONDYS 53

NDA NDA211970

Feb 11, 2021

VYONDYS 53

NDA NDA211970

Jun 4, 2024

VYONDYS 53

NDA NDA211970

Jun 4, 2024

VYONDYS 53

NDA NDA211970

Sources

References

[1]
ClinicalTrials.gov — 3 trial records for Golodirsen. U.S. National Library of Medicine, National Institutes of Health.
[2]
ChEMBL — Golodirsen compound record. European Bioinformatics Institute (EMBL-EBI).
[3]
Open Targets Platform — Drug–disease associations for Golodirsen. EMBL-EBI & Wellcome Sanger Institute.
[4]
OpenFDA — FDA approval records (NDA NDA211970). U.S. Food & Drug Administration.
[5]
FDA Orange Book — Patent and exclusivity data for Vyondys 53. U.S. Food & Drug Administration.
[6]
SEC EDGAR — Sarepta Therapeutics company filings. U.S. Securities and Exchange Commission.

Details

Drug Info

Quick Facts

Mechanism of Action: Exon 53 Skipping ASO[2]
Modality: RNA therapy[2]
Highest Phase: Approved[1]
Company: Sarepta Therapeutics

Patent Landscape[5]

Patent Cliff

Jun 28, 2028

Earliest patent expiration — generic entry possible after this date

Patent #Expires

RE47691Jun 28, 2028

Vyondys 53

Status by Indication[3][1]

Competitive Landscape

Trials by Indication[1]

Duchenne Muscular Dystrophy2 trials

Observational Study1 trial

FDA Approval Timeline[4]

References

Drug Info

Quick Facts

Patent Landscape[5]

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