Who Is It For

Who can be treated with EXONDYS 51 (eteplirsen)?


EXONDYS 51 is used to treat Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation in the dystrophin gene that can be treated by skipping exon 51. EXONDYS 51 was approved under accelerated approval. Accelerated approval allows for drugs to be approved based on a marker that is considered reasonably likely to predict a clinical benefit.
EXONDYS 51 treatment increased the marker, dystrophin, in skeletal muscle in some patients. Verification of a clinical benefit may be needed for EXONDYS 51 to continue to be approved. 

Do you want to know if your child can be treated with EXONDYS 51? This information will help prepare you to have a more informed discussion with your doctor.

Micah

Meet Micah, age 8
Deletion of exon 50

Know your mutation.

If you haven’t already done so, get a genetic test to identify your child’s genetic mutation; your doctor or a genetic counselor can interpret the results to determine if the mutation is one of the 13% that are amenable to treatment with EXONDYS 51. Genetic test results may also help your doctor make other disease management decisions.

See More on Amenability
Liam
Test

Understand test results.

When your child’s test results come back, it’s sometimes confusing to know what you’re reading. We’ll break it down and provide information you can use to talk to your child’s doctor about eligibility for EXONDYS 51.

See a Sample Test Result

Create your personalized Doctor Discussion Guide.

Answer a few simple questions to generate a custom discussion guide and be prepared for your child’s next doctor appointment. The guide may help you discuss important topics with your doctor so you can understand if EXONDYS 51 is a possibility for your child.

Create a Guide
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WHAT IS EXONDYS 51 (eteplirsen)?

EXONDYS 51 is used to treat Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation in the dystrophin gene that can be treated by skipping exon 51. EXONDYS 51 was approved under accelerated approval. Accelerated approval allows for drugs to be approved based on a marker that is considered reasonably likely to predict a clinical benefit. EXONDYS 51 treatment increased the marker, dystrophin, in skeletal muscle in some patients. Verification of a clinical benefit may be needed for EXONDYS 51 to continue to be approved.

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IMPORTANT RISK INFORMATION

Allergic reactions, including wheezing, chest pain, cough, rapid heart rate, and hives have occurred in patients who were treated with EXONDYS 51. Seek immediate medical care if signs and symptoms of allergic reactions occur.

Side effects that happened at least 25% more often in 8 patients treated with EXONDYS 51 by intravenous infusion than in 4 patients treated with an inactive intravenous infusion were problems with balance (38%, 0%), vomiting (38%, 0%), and skin irritation (25%, 0%). The most common side effects were problems with balance and vomiting.

The most common side effects seen in greater than 10% of patients receiving EXONDYS 51 (N=163) in other clinical trials were headache, cough, rash, and vomiting. 

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 EXONDYS 51 (eteplirsen).