What Is VYONDYS 53?

An exon-skipping therapy used to treat Duchenne.

VYONDYS 53 is used to treat patients with Duchenne muscular dystrophy (DMD) who have a confirmed mutation in the dystrophin gene that can be treated by skipping exon 53.

This indication is approved under accelerated approval based on an increase in dystrophin production in skeletal muscle observed in patients treated with VYONDYS 53. Continued approval for this indication may be contingent upon verification of a clinical benefit in confirmatory trials.

Meet Nicholas, age 17
Amenable to exon 53 skipping

How does it work?

VYONDYS 53 is an exon-skipping therapy that helps the body make a shorter form of the dystrophin protein. Find out more about VYONDYS 53 and the results from its clinical studies.

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A lack of dystrophin protein

Exon-skipping technologies strive to address the underlying issue with Duchenne—a lack of the protein dystrophin. Many people with Duchenne have a genetic mutation in which one or more exons in the dystrophin gene are missing. This causes errors in the instructions for making dystrophin, leaving the body unable to produce the protein.

Exon skipping tells the body to hide an exon so the whole section can be skipped over and the remaining exons can fit together. VYONDYS 53 is intended to bind with exon 53. VYONDYS 53 helped some boys with DMD amenable to skipping exon 53 to make a shorter form of dystrophin protein.

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Informed decisions.

There are certain risks and side effects associated with VYONDYS 53. As with any medication, you should discuss risks and side effects with your child's doctor.

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WHAT IS VYONDYS 53 (golodirsen)?

VYONDYS 53 is used to treat patients with Duchenne muscular dystrophy (DMD) who have a confirmed mutation in the dystrophin gene that can be treated by skipping exon 53.

IMPORTANT RISK INFORMATION

Allergic reactions, including rash, fever, itching, hives, and inflammation and/or peeling of the skin have occurred in patients who were treated with VYONDYS 53. Seek immediate medical care if signs and symptoms of allergic reactions occur.

Damage to the kidneys was seen in animals who received golodirsen. Although damage to the kidneys was not seen in clinical studies with VYONDYS 53, potentially fatal kidney damage has occurred with other drugs that work in a similar way. Your doctor may recommend urine and blood testing before starting treatment followed by urine testing every month and a blood test every 3 months to monitor your kidneys.

IMPORTANT RISK INFORMATION

Adverse reactions that have occurred in at least 20% of patients treated with VYONDYS 53 and more often than in patients who received an inactive intravenous (IV) infusion were headache (41%, 10%), fever (41%, 14%), fall (29%, 19%), pain in the abdomen (27%, 10%), infection of the nose and throat (27%, 14%), cough (27%, 19%), vomiting (27%, 19%), and nausea (20%, 10%).

Other adverse reactions that occurred in greater than 5% of patients treated with VYONDYS 53 and more often than in patients who received an inactive IV infusion were pain at the IV site, back pain, pain, diarrhea, dizziness, stretch or tear in a ligament, bruising, flu, pain in the mouth and throat, stuffy or runny nose, scrapes or scratches of the skin, ear infection, seasonal allergy, fast heartbeat, reactions related to the IV catheter site, constipation, and broken bones.

You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch or call 1-800-FDA-1088. You may also report side effects to Sarepta Therapeutics at 1-888-SAREPTA (1-888-727-3782).

Please see the full Prescribing Information for VYONDYS 53 (golodirsen).