How It Works

Treatment with VYONDYS 53 (golodirsen).

A lack of dystrophin causes muscle cells to become damaged and weakened over time. 
VYONDYS 53 uses exon-skipping technology to allow the body to make a shorter form of the dystrophin protein in some patients by skipping over a specific exon on the dystrophin gene.


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Amenable to exon 53 skipping

Amenable

a•me•na•ble

əˈmēnəb(ə)l, əˈmenəb(ə)l/

(of a thing) capable of being acted upon in a particular way;

susceptible to.

The Duchenne patients genetic test identified that he had a mutation that could be treated by VYONDYS 53. His doctor said he was amenable to treatment with the exon-skipping therapy.”

What is exon skipping?

Duchenne is caused by a genetic mutation in the dystrophin gene. Most commonly, one or more exons (parts of the gene) are missing, causing errors in the instructions for making dystrophin. This results in the body not being able to produce enough—or any—working dystrophin protein. The goal of exon skipping is to allow the body to make a shorter form of the dystrophin protein. This video will show you how.


How exon skipping works.

Understanding the dystrophin gene.


The dystrophin gene is made up of exons (portions of a gene) that are linked together to provide instructions for making dystrophin—a protein our muscles need to work properly. Without dystrophin, muscle cells become damaged and weaken over time.

How exons connect.


With 79 exons, the dystrophin gene is the largest in the body. Think of the exons on the dystrophin gene like toy train cars, each with a special connection that allows one car to connect to another. In order for all the cars to move together as a train, the connections between cars must match—for example, circle-to-circle and square-to-square.

When an exon is missing.


Here we can see that cars, or exons, 50, 51, and 52, are missing. The result is that cars 49 and 53 are not able to connect because their connectors are different shapes and don’t fit together. In the dystrophin gene, this missing exon would prevent the body from being able to read the instructions for making the dystrophin protein.

Skipping over exons.


By moving certain exons—or cars—aside, we can “skip over” them to find a car with the right connector. The new train would be shorter, but the cars are connected. Just as we skipped over a train car, VYONDYS 53 is designed to skip over an exon.

 

 

The result: A shorter form of dystrophin.


VYONDYS 53 works using exon-skipping technology. In some boys, weekly infusions with VYONDYS 53 have been shown to help the body make a shorter form of the dystrophin protein. Watch the video.

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Clinical studies of VYONDYS 53 tested whether exon skipping happened on the dystrophin gene of boys treated with the drug. In those studies, exon skipping occurred in all 25 evaluated study participants. Boys who received VYONDYS 53 had variable responses in the amount of dystrophin production.

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Related FAQs

Who can take VYONDYS 53?

Duchenne patients who receive VYONDYS 53 must have a genetic test that shows a mutation in the dystrophin gene that can be treated by skipping exon 53. Your child's doctor is best equipped to determine if your child’s mutation is amenable to treatment with VYONDYS 53. We’ve developed a Doctor Discussion Guide to help you start that important conversation.

Has VYONDYS 53 been tested?

Yes. VYONDYS 53 has been studied in clinical trials. See the results from clinical trials.

 

 

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.

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.

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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

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.

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).