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Vyondys 53

Pronunciation: vee-yon-deez
Generic name: golodirsen
Other brand names of golodirsen include: Vyondys, Vyondys 53
Dosage form: injection for intravenous infusion (100 mg/2 mL)
Drug class: Miscellaneous uncategorized agents

Medically reviewed by Carmen Pope, BPharm. Last updated on May 20, 2025.

What is Vyondys 53?

Vyondys 53 is used for Duchenne muscular dystrophy (DMD) in children and young adults with a certain gene mutation. It is an exon-skipping treatment given 1 time a week via an intravenous infusion by a healthcare provider.

Vyondys 53 (golodirsen) gained FDA approval on December 12, 2019, under the accelerated approval designation. Continued approval may be contingent upon verification of a clinical benefit in clinical trials.

How does Vyondys 53 work?

Vyondys' mechanism of action uses exon-skipping technology to skip over exon 53, which allows the body to make a shorter form of a protein called dystrophin, needed for muscle integrity. People with DMD have a genetic mutation in the gene that encodes for this protein, which means they cannot produce enough, or any, working copies of this protein. Without this protein, muscle cells become damaged and weaken with time.

By skipping over exon 53, Vyondys allows different exons to join together to produce a dystrophin protein that is shorter but has some function. Clinical trials report exon-skipping as confirmed by muscle biopsy in 25 of 25 study participants. After 48 weeks of treatment, participants showed an average increase of nearly 16 times their baseline level of dystrophin.

Vyondys 53 belongs to the drug class called antisense oligonucleotides.

Side effects

The most common side effects of Vyondys are:

Other common side effects of Vyondys are:

Serious side effects and warnings

Vyondys 53 can cause the following serious side effects:

This is not a complete list of side effects, and others may occur. Call your doctor for medical advice about side effects. You may report side effects to the FDA at 1-800-FDA-1088.

Before receiving this medicine

Before receiving Vyondys 53, tell your healthcare provider if you:

How will I receive Vyondys 53?

Vyondys is administered as an IV infusion (an infusion into your vein) 1 time a week by a healthcare provider.

Hypersensitivity reactions can occur when Vyondys 53 is administered. If this happens, then the infusion may need to be slowed or interrupted.

Your kidney function may need to be checked while receiving treatment with Vyondys 53.

Dosing information

Check serum cystatin C, urine dipstick, and urine protein-to-creatinine ratio before starting Vyondys 53.

What happens if I miss a dose?

Call your doctor for instructions if you miss an appointment for your Vyondys 53.

What should I avoid while receiving Vyondys 53?

Follow your doctor's instructions about any restrictions on food, beverages, or activity.

What other drugs will affect Vyondys 53?

Other drugs may affect Vyondys 53, including prescription and over-the-counter medicines, vitamins, and herbal products. Tell your doctor about all your current medicines and any medicine you start or stop using.

Does Vyondys 53 interact with my other drugs?

Enter medications to view a detailed interaction report using our Drug Interaction Checker.

Vyondys 53 ingredients

Active ingredients: golodirsen 100 mg (50 mg/mL)

Inactive ingredients: Each milliliter contains 0.2 mg potassium chloride, 0.2 mg potassium phosphate monobasic, 8 mg sodium chloride, and 1.14 mg sodium phosphate dibasic, anhydrous, in water for injection. May contain hydrochloric acid or sodium hydroxide to adjust pH.

Formulated as an isotonic phosphate-buffered saline solution with an osmolality of 260 to 320 mOsm and a pH of 7.5.

Available as a 100 mg/2 mL (50 mg/mL) in a single-dose vial for injection.

Manufacturer

Vyondys 53 is manufactured by Sarepta Therapeutics, Inc. Their headquarters are located in Cambridge, Massachusetts, USA. Sarepta Therapeutics specializes in the development of RNA-targeted therapeutics, with a focus on treatments for rare diseases, particularly Duchenne muscular dystrophy (DMD).

Popular FAQ

What are the new drugs for DMD (Duchenne muscular dystrophy)?

The new drugs approved by the FDA for the treatment of Duchenne Muscular Dystrophy (DMD) are Agamree, Amondys 45, Duvyzat, Elevidys, Emflaza, Exondys 51, Viltepso, and Vyondys 53. They include classes such as antisense oligonucleotides, glucocorticoid (corticosteroids), a gene therapy and a histone deacetylase (HDAC) inhibitor. Continue reading

How does Vyondys 53 work?

Vyondys 53 is an antisense oligonucleotide that works by binding to exon 53 of dystrophin pre-mRNA, which results in this exon being skipped over during mRNA processing. Skipping of exon 53 allows cells to join different exons together to produce a dystrophin protein that is shorter but has some function. Continue reading

How long does it take for Vyondys 53 to work?

Results from a small phase I/II clinical trial show that treatment with Vyondys 53 increases dystrophin protein levels within 48 weeks of starting treatment. Continue reading

How is Vyondys 53 administered?

Vyondys 53 (golodirsen) is administered by intravenous (IV) infusion once a week by a healthcare provider. Continue reading

Further information

Always consult your healthcare provider to ensure the information displayed on this page applies to your personal circumstances.