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 41 aso programs targeting Duchenne Muscular Dystrophy. It is currently in Approved.

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 DystrophyApproved

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

Duvyzat

Italfarmaco

Phase 3

2 8

Inspra

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

Phase 3

2 9

Bystolic

VVitae Pharmaceuticals Inc., an Allergan affiliate

Phase 3

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

Terminated· Sarepta Therapeutics logo

Sarepta Therapeutics·Phase 3·NCT03532542·171 enrolled

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

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

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]

Related News