NDAINTRAVENOUSSOLUTIONPriority Review
Approved
Dec 2019
Lifecycle
LOE Approaching
Competitive Pressure
15/100
Clinical Trials
5
Data completeness
3/8 — Basic
Priority Review
Drug data last refreshed 18h ago · AI intelligence enriched 1w ago
AI summarySynthesized from FDA + DailyMed + ClinicalTrials.gov data
VYONDYS 53 (golodirsen) is an antisense oligonucleotide administered intravenously for Duchenne muscular dystrophy (DMD). It works by modulating dystrophin pre-mRNA splicing to allow production of internally truncated but functional dystrophin protein in patients with specific DMD mutations amenable to exon 53 skipping.
LOE Approaching2.0 years to LOE
Product is in late-stage commercial maturity with LOE approaching in 2028; teams should expect transition planning and focus on maximizing remaining exclusivity period.
Mechanism of Action
Pharmacologic Class:
Antisense Oligonucleotide
Indications
Indication data is being enriched from DailyMed and FDA labeling. Check back soon for approved therapeutic uses.
Clinical Trials (5)
An Open-Label Study of Golodirsen in Non-Ambulant Patients With Duchenne Muscular Dystrophy
Started Oct 2020
2 enrolled
Duchenne Muscular Dystrophy
A 48-Week, Open Label, Study to Evaluate the Efficacy and Safety of AMONDYS 45, EXONDYS 51, VYONDYS 53 in Subjects With DuchenneMuscular Dystrophy Carrying Eligible DMD Duplications.
Started Feb 2020
3 enrolled
Duchenne Muscular Dystrophy
A Long-term Observational Study Evaluating Eteplirsen, Golodirsen, or Casimersen in Routine Clinical Practice
Started Jan 2019
300 enrolled
Duchenne Muscular Dystrophy
An Extension Study to Evaluate Casimersen or Golodirsen in Patients With Duchenne Muscular Dystrophy
Started Aug 2018
171 enrolled
Duchenne Muscular Dystrophy
Study of SRP-4045 (Casimersen) and SRP-4053 (Golodirsen) in Participants With Duchenne Muscular Dystrophy (DMD)
Started Sep 2016
228 enrolled
Duchenne Muscular Dystrophy
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Data Sources
- Drug label: DailyMed / FDA
- Clinical trials: ClinicalTrials.gov
- Patent data: FDA Orange Book
- Spending data: CMS Medicare
The information on this page is for informational purposes only and should not be used as a substitute for professional medical advice. Drug information is sourced from FDA, DailyMed, and other government databases. Adverse event data from FAERS does not establish causation. Always consult a healthcare professional for medical decisions.