VYONDYS 53
VYONDYS 53 (golodirsen) is an antisense oligonucleotide indicated for the treatment of Duchenne muscular dystrophy (DMD). It is specifically used for patients who have a confirmed mutation of the DMD gene that is amenable to exon 53 skipping. The drug received accelerated approval based on an observed increase in dystrophin production in skeletal muscle, though continued approval may be contingent upon verification of clinical benefit in confirmatory trials.
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
- 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
Drugs Similar to VYONDYS 53
FDA-approved drugs for similar conditions. Compare mechanisms and indications to understand treatment alternatives.
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
ProIndications & Usage
FDA Label (PDF)VYONDYS 53 is indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 53 skipping. This indication is approved under accelerated approval based on an increase in dystrophin production in skeletal muscle observed in patients treated with VYONDYS 53 [see Clinical Studies ] . Continued approval for this indication may be contingent upon verification of a clinical benefit in confirmatory trials. VYONDYS 53 is an antisense oligonucleotide indicated for the treatment of Duchenne muscular dystrophy (DMD) in pa...
VYONDYS 53 Patents & Exclusivity
Patents (1 active)
Exclusivity
Want competitive intelligence?
See who's developing similar drugs and track their progress
Data Sources
Data sourced from official FDA and NIH databases. Click links to verify on original sources.