Quick answers
- What is Golodirsen used for?
- The FDA-approved label (Vyondys 53, Sarepta Therapeutics, Inc., March 13, 2025) says: “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 […]. …” This page is not medical advice. Talk to your doctor or pharmacist before you start, stop or change any medicine.
- Does Golodirsen have a boxed warning?
- No. None of the 1 FDA labels we found for this medicine has a boxed warning.
- Is there a shortage of Golodirsen?
- No current shortage is listed by the FDA for the products of this medicine (FDA data from October 30, 2026).
- Has Golodirsen been recalled?
- No recall linked to the products of this medicine appears in the FDA enforcement reports we use.
- Who makes Golodirsen?
- 1 company lists prescription products of Golodirsen in the FDA NDC Directory, including Sarepta Therapeutics, Inc..
What Golodirsen is used for
Quoted from the “Indications and Usage” section of the FDA-approved label.
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 […]. …
— FDA label: Vyondys 53, Sarepta Therapeutics, Inc., effective March 13, 2025. Read the full label on DailyMed →
This page is not medical advice. Talk to your doctor or pharmacist before you start, stop or change any medicine.
Boxed warning
A boxed warning (“black box”) is the FDA’s strongest warning on a drug label, for serious or life-threatening risks.
The label quoted above has no boxed warning.
Forms and how it is given
| Dosage forms | Injection |
|---|---|
| Route | Intravenous |
| Prescription products | 1 in the FDA NDC Directory |
| Brand names | Vyondys 53 |
Companies that list this medicine
Labelers in the FDA NDC Directory: the company named on the package (maker or distributor), with the number of prescription products.
- Sarepta Therapeutics, Inc.1 product
FDA approval history
- NDA: New Drug Application: how a new (usually brand-name) medicine is approved.
| Application | Sponsor | Approved | Later changes approved |
|---|---|---|---|
| NDA211970 VYONDYS 53 | Sarepta Theraps Inc | December 12, 2019 | 3, latest June 4, 2024 2024: Labeling · 2024: Labeling · 2021: Labeling |
Shortage status
The FDA drug shortage list does not show these products.
Recalls
No recall linked to these products (by their NDC code) appears in the FDA enforcement reports.
Official identifiers
| RxCUI (RxNorm) | 2267211, 2267216 |
|---|---|
| SPL set ID of the quoted label | 35c227d1-5b24-44b0-b5d3-f0f6b1c46bd5 |
| FDA labels (SPL) | 1 |
| FDA applications | NDA211970 |
| Active ingredients (FDA) | GOLODIRSEN |
Sources
- FDA-approved label on DailyMed (National Library of Medicine) — Vyondys 53, Sarepta Therapeutics, Inc., effective March 13, 2025; via openFDA, FDA data exported October 30, 2026, retrieved October 31, 2026
- NDC Directory (openFDA) — U.S. Food and Drug Administration (FDA), FDA data exported October 30, 2026, retrieved October 31, 2026
- Drugs@FDA (openFDA) — U.S. Food and Drug Administration (FDA), FDA data exported October 30, 2026, retrieved October 31, 2026
- Drug recall enforcement reports (openFDA) — U.S. Food and Drug Administration (FDA), FDA data exported October 27, 2026, retrieved October 31, 2026
- Drug shortages (openFDA) — U.S. Food and Drug Administration (FDA), FDA data exported October 30, 2026, retrieved October 31, 2026
openFDA data is provided by the FDA as is; it may be incomplete. Always rely on the label that comes with your medicine. FDA Enforcement Report search →