Pronunciation: vee-yon-deez
Generic name: golodirsen
Other brand names of golodirsen include: Vyondys
Dosage form: injection for intravenous infusion
Drug class: Miscellaneous uncategorized agents

Medically reviewed by Carmen Pope, BPharm. Last updated on Mar 11, 2025.

What is Vyondys 53?

Vyondys 53 is used to treat Duchenne muscular dystrophy (DMD) in patients with a confirmed mutation of the DMD gene that is amenable to exon 53 skipping.  It is given weekly as an intravenous infusion that takes about 35 to 60 minutes.

Your healthcare provider will conduct a genetic test to identify if you or your child is one of the estimated 8% of people with DMD who are suitable for treatment with Vyondys 53.

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 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 which is needed for muscle integrity. People with DMD have a genetic mutation in the gene that encodes for this protein, which means they are not able to 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.

Vyondys 53 belongs to the drug class called antisense oligonucleotides.

Vyondys 53 side effects

The most common side effects of Vyondys 53 are:

• Headache
• Fever
• Fall
• Abdominal pain
• Nose or throat infection
• Cough
• Vomiting
• Nausea.

Other side effects include:

• Pain at the injection site  or reactions related to the IV catheter site
• Back pain, mouth or throat pain, or general pain
• Diarrhea or constipation
• Dizziness
• Flu, stuffy/runny nose, ear infections, seasonal allergies
• Bruising, skin scratches or scrapes, ligament stretches or tears, broken bones
• Fast heartbeat.

Serious side effects and warnings

Hypersensitivity reactions, including anaphylaxis, rash, fever, itch, hives, dermatitis, and skin exfoliation have occurred in patients who were treated with Vyondys 53. If these occur during the infusion, notify your healthcare provider immediately. Get emergency medical help if you have signs of an allergic reaction: hives, itching, rash, blistering or peeling; fever; difficult breathing; swelling of your face, lips, tongue, or throat.

Vyondys may cause damage to your kidneys. Your healthcare provider will monitor your kidney function during treatment. Call your doctor at once if you have:

• pink, brown, or red urine;
• foamy urine; or
• swelling in your face, hands, feet, or stomach.

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 FDA at 1-800-FDA-1088.

Related/similar drugs

Before receiving this medicine

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

• Have ever had kidney problems
• Have had an allergic reaction to Vyondys 53
• Are pregnant or intending to become pregnant
• Are breastfeeding.

How is Vyondys 53 given?

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

• Your doctor will perform blood tests to ensure you or your child are suitable for Vyondys 53 and do not have any conditions that would prevent you from safely receiving it.
• Topical anesthetic cream can be applied to the site where the infusion will be administered if it is needed to help numb the area.
• Vyondys 53 must be given slowly, and the infusion can take up to 60 minutes to complete.

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 you are receiving treatment with Vyondys 53.

Dosing information

The recommended dose of Vyondys 53 is 30 mg per kg of body weight given once weekly.

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.

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.

The product 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.

Who makes Vyondys 53?

Vyondys 53 is manufactured by Sarepta Therapeutics, Inc. Their headquarters are located in Cambridge, Massachusetts, USA.

Sarepta Therapeutics is a biopharmaceutical company that specializes in the development of RNA-targeted therapeutics, with a focus on treatments for rare diseases, particularly Duchenne muscular dystrophy (DMD).

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Further information

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

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