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About EXONDYS 51

How will I know if my child can be treated with EXONDYS 51?

A genetic test is the only way for your child's doctor to identify your child's mutation and determine if the mutation is amenable to treatment with EXONDYS 51. Learn how genetic mutations are identified.

Who is EXONDYS 51 for?

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. Learn how gene mutations are identified.

 

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 that has not been proven. 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.

Is the treatment effective?

Weekly infusions of EXONDYS 51 helped the body make a shorter form of the dystrophin protein in some boys. View the results from clinical studies.

 

EXONDYS 51 is used to treat Duchenne 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 that has not been proven. 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.

Genetic Testing

Why is genetic testing important?

A genetic test will confirm a diagnosis of Duchenne and allow your child’s doctor to identify the genetic mutation so treatment options can be considered and to assist with family planning. Understanding genetic testing.

What does “amenable” mean?

Amenability describes the potential for your child to be treated with exon-skipping therapy. EXONDYS 51 is only for those whose Duchenne muscular dystrophy is the result of a genetic mutation amenable to exon 51 skipping. Find out more about genetic testing for Duchenne.

Where do I get a genetic test?

If your child has been diagnosed with Duchenne, you can request a genetic test through your child’s doctor. Once you have the results, your doctor or a genetic counselor can interpret them for you. Find out more about genetic testing for Duchenne.

We have a genetic test. How do I know if my child can be treated with EXONDYS 51?

A doctor will need to interpret the test results to consider appropriate treatment options. Learn more about deletions.

What is a genetic mutation?

A mutation is a change in a person's DNA. Mutations range in size from a small (a single rung on a ladder) to a large segment of DNA. Every mutation causes a different effect on our bodies. Learn more about the role of genetics in Duchenne.

What is a deletion?

A deletion is a type of mutation where genetic material is missing. Duchenne is caused by a mutation on the dystrophin gene; the mutation may be a deletion, duplication, or other change in the gene.  Learn about genetic testing for Duchenne.

My child has a deletion and is missing exon 51. Can they take EXONDYS 51?

Please discuss your child's genetic test results and treatment options with your child's doctor. For EXONDYS 51 to work, exon 51 must be present in the dystrophin gene. Your child's doctor or a genetic counselor is the best person to help you understand genetic testing. Explore what genetic test results can tell you.

Why is a Duchenne diagnosis important?

Because Duchenne is a progressive disease, it’s important for your child's doctor to confirm a diagnosis and identify the specific genetic mutation to guide your child's care and treatment. More about the steps to diagnosis.

EXONDYS 51 Treatment

Who can take EXONDYS 51?

Duchenne patients who receive EXONDYS 51 must have a genetic test that shows a mutation in the dystrophin gene that can be treated by skipping exon 51. Your child's doctor is best equipped to determine if your child’s mutation is amenable to treatment with EXONDYS 51. We’ve developed a Doctor Discussion Guide to help you start that important conversation.

How is EXONDYS 51 given?

EXONDYS 51 therapy is a weekly intravenous (IV) infusion that is always given by a healthcare provider. The medicine is usually infused over 35-60 minutes. Learn more about planning for treatment.

Where will I get my infusion?

Infusions can be given at an infusion center, doctor's office, or your home. Discuss your options with your doctor—there are many factors to consider when deciding what’s best for your family.

What happens if I miss an infusion?

Talk to your healthcare provider. If you miss a dose of EXONDYS 51, it may be administered as soon as possible after the scheduled time.

Should I continue taking my other medications while on EXONDYS 51?

You should talk to your doctor about all the medications you are taking. Your doctor is the best person to advise you about your medicines.

How much EXONDYS 51 will I receive?

The amount of EXONDYS 51 is based upon how much you weigh. The recommended dosage is 30 milligrams per kilogram of body weight, once weekly.

About SareptAssist™

How do I get started on EXONDYS 51?

When your child is prescribed EXONDYS 51, we’ll connect you with a SareptAssist dedicated Case Manager. They will provide support to get you started on treatment and throughout your journey—from helping you understand insurance benefits to providing information on treatment locations. How SareptAssist can help.

What is SareptAssist?

SareptAssist is our patient support program. Families are connected with dedicated Case Managers who provide information and support to help them start and stay on treatment with EXONDYS 51. How SareptAssist can help.

What support is available for people being treated with EXONDYS 51?

We developed SareptAssist, our patient support program, to help patients start and stay on therapy. You’ll have support at every step—from managing insurance approvals to coordinating drug delivery. How SareptAssist can help.

How can I connect with other families of children with Duchenne?

Several advocacy organizations offer Duchenne patients and families the opportunity to come together at events throughout the year. Find out more about Duchenne advocacy groups.

Will my insurance cover EXONDYS 51?

Once your child is prescribed EXONDYS 51, you may enroll in SareptAssist, our patient support program. Your Case Manager will start a benefits investigation on your current insurance plan and can help explain details about your coverage. How SareptAssist can help.

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 that has not been proven. 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.

IMPORTANT RISK INFORMATION

Allergic reactions, including rash, hives, fever, flushing, cough, difficulty breathing, wheezing, and low blood pressure 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.

In patients who received EXONDYS 51 for up to 4 years in clinical studies, the following side effects were reported in at least 10% of patients and occurred more often than at the same dose of EXONDYS 51: vomiting, bruising, scratches, joint pain, rash, IV site pain, and infections of the upper airways.

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).

READ MORE

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 that has not been proven. 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.

IMPORTANT RISK INFORMATION

Allergic reactions, including rash, hives, fever, flushing, cough, difficulty breathing, wheezing, and low blood pressure 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.

In patients who received EXONDYS 51 for up to 4 years in clinical studies, the following side effects were reported in at least 10% of patients and occurred more often than at the same dose of EXONDYS 51: vomiting, bruising, scratches, joint pain, rash, IV site pain, and infections of the upper airways.

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).