About VYONDYS 53

Weekly infusions of VYONDYS 53 (golodirsen) have been shown to help the body make a shorter form of the dystrophin protein in some boys.

Understanding VYONDYS 53.

VYONDYS 53 is a treatment for Duchenne muscular dystrophy (DMD). It uses a technology called exon skipping to help the body make a shorter form of the dystrophin protein. VYONDYS 53 is given in a once-weekly infusion.

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.

 

Results from VYONDYS 53 clinical studies.

Researchers conducted clinical trials of VYONDYS 53 to review different aspects of the medicine, including whether it triggers skipping of exon 53 on the dystrophin gene. They also analyzed the amount of dystrophin produced, along with its safety profile. VYONDYS 53 was studied in boys who had a confirmed mutation in the dystrophin gene that could be treated by skipping exon 53. Learn what this means. 

 

Designed to skip exon 53.


One clinical study looked at whether exon skipping occurred on the dystrophin gene of boys treated with VYONDYS 53. In clinical studies, exon skipping occurred in all 25 evaluated study participants.

 

Dystrophin levels increased in some clinical trial participants.

A study of 25 boys with Duchenne muscular dystrophy compared the level of dystrophin in their bodies before the first infusion (baseline) of VYONDYS 53 with the level of dystrophin after 48 weeks of treatment with VYONDYS 53. Weekly infusions of VYONDYS 53 helped the body make a shorter form of the dystrophin protein. The amount of dystrophin produced varied in the study.

 

Dystrophin levels measured by western blot can be strongly affected by differences in how the sample is processed, the reference material used, and the quantification method.

Comparing dystrophin results from other methods of measurement requires standard reference material and additional studies.

 

 

What do the results tell us?

 

 

 


What about risks? Researchers assessed the risks and tolerability of VYONDYS 53. As with any medication, you should discuss the risks and side effects associated with VYONDYS 53 with your doctor.

Review Risks and Side Effects

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).