VYONDYS 53 (golodirsen)
VYONDYS 53 is indicated for the treatment of Duchenne Muscular Dystrophy.
How VYONDYS 53 Works
Golodirsen is designed to bind to exon 53 of dystrophin pre-mRNA, resulting in the exclusion of this exon during mRNA processing. This exon skipping is intended to allow for the production of an internally truncated dystrophin protein in patients with specific genetic mutations. By facilitating the production of this protein, the drug targets the underlying genetic mutation in patients amenable to exon 53 skipping.
Details
- Status
- Prescription
- First Approved
- 2019-12-12
- Patent Cliff
- 2028
- Routes
- INTRAVENOUS
- Dosage Forms
- SOLUTION
VYONDYS 53 Approval History
What VYONDYS 53 Treats
1 indicationsVYONDYS 53 is approved for 1 conditions since its original approval in 2019. These indications span multiple therapeutic areas including oncology, immunology, and more.
- Duchenne Muscular Dystrophy
VYONDYS 53 Competitive Set
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Direct competitors
Same target(s) AND same indication — head-to-head.
Indication competitors
Same indication, different mechanism — what else might this patient receive?
Filters applied: drops same-active-ingredient (505(b)(2) reformulations), route-mismatch (topical vs systemic), and cross-therapeutic-area matches in same-indication rings.
What's emerging in VYONDYS 53's indications
See all emerging drugs →Phase 3 candidates targeting molecules with no FDA-approved drug, in indications VYONDYS 53 treats. First-in-class if their pivotal trials read out positive.
Drugs Similar to VYONDYS 53
3 of 9FDA-approved drugs for similar conditions. Compare mechanisms and indications to understand treatment alternatives.
Clinical Trial Registry
2 trials| Trial | Sponsor ID | Phase | Status | Title |
|---|---|---|---|---|
| NCT03532542 results posted | 4045-302 2017-004625-32 | Ph 3 | terminated | An Extension Study to Evaluate Casimersen or Golodirsen in Patients With Duchenne Muscular Dystrophy |
| NCT04179409 results posted | SRPT-Dup-US-001 | Ph 2 | completed | 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. |
Active Pipeline
Ongoing clinical trials by development phase
Key Completed Trials
Completed studies with published results, ranked by significance
Trial Timeline
Full development history with FDA approval milestones
Understanding FDA Approval Types
| Count | Type | What it means |
|---|---|---|
| - | ORIG | Original approval - drug first enters market |
| - | SUPPL - Efficacy | New indication (new disease/condition approved) |
| - | SUPPL - Labeling | Label text changes (warnings, dosing updates) |
| - | SUPPL - Manufacturing | Production changes (new facility) |
| - | SUPPL - Chemistry | Formulation changes (new dosage strength) |
Green lines in the timeline show ORIG and Efficacy approvals - the clinically meaningful milestones.
VYONDYS 53 FDA Label Details
Indications & Usage
FDA Label (PDF)VYONDYS 53 is indicated for the treatment of Duchenne Muscular Dystrophy.
VYONDYS 53 Patents & Exclusivity
Patents (1 active)
Exclusivity
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Deep insights for VYONDYS 53
Revenue Insights
- • Quarterly revenue tracking
- • Historical trend analysis
Patent Timeline
- • Cliff: 2028
- • 1 active patents
Trial Analysis
- • Clinical trial tracking
- • Development stage analysis
Competitive Landscape
- • 9 similar drugs
- • Same target/indication analysis
Full approval history • All patents • Revenue trends • Competitor analysis
Data Sources
Data sourced from official FDA and NIH databases. Click links to verify on original sources.