eteplirsen

What is the most important information I should know about eteplirsen?

Before your child receives eteplirsen, tell the doctor about all your child's medical conditions or allergies, and all the medicines the child uses.

What is eteplirsen?

Eteplirsen works by helping the body produce a protein called dystrophin. Dystrophin is a protein that helps stabilize the structure of muscle tissue and is necessary for proper muscle development and function. Without dystrophin, muscle fibers can become weak and easily damaged. This can affect voluntary movement such as walking.

Children with Duchenne muscular dystrophy have a mutation of a gene that would normally cause the body to produce dystrophin.

Eteplirsen is used in children with Duchenne muscular dystrophy (DMD). Eteplirsen is not a cure for DMD, but this medicine may lessen muscle weakness and muscle wasting caused by this disease.

Eteplirsen was approved by the US Food and Drug Administration (FDA) on an "accelerated" basis. In clinical studies, some children responded to this medicine. However, further studies are needed to determine if eteplirsen is generally effective in treating DMD.

Eteplirsen may also be used for purposes not listed in this medication guide.

What should I discuss with my healthcare provider before receiving eteplirsen?

Before your child receives eteplirsen, tell the doctor about all your child's medical conditions or allergies, and all the medicines the child uses.

Although eteplirsen has been used mainly in children, it is not known whether this medicine could be harmful if used during pregnancy or while nursing.

How is eteplirsen given?

Eteplirsen is given as an infusion into a vein. A healthcare provider will give you this injection.

To make the injection more comfortable, a numbing medicine may be applied to the skin area where the IV needle will be placed.

Eteplirsen is given once per week. This medicine must be injected slowly, and the infusion can take up to 1 hour to complete.

Tell your caregivers if you feel itchy, warm, light-headed, or have chest tightness or trouble breathing when eteplirsen is injected.

Tell your doctor if your child has any changes in weight. Eteplirsen doses are based on weight, and any changes may affect the dose.

What happens if I miss a dose?

What happens if I overdose?

What should I avoid while receiving eteplirsen?

Follow your doctor's instructions about any restrictions on food, beverages, or activity.

What are the possible side effects of eteplirsen?

Common side effects may include:

• pain, bruising, itching, skin rash, or other irritation where the medicine was injected;
• vomiting;
• joint pain;
• problems with balance; or
• cold symptoms such as stuffy nose, sneezing, sore throat.

This is not a complete list of side effects and others may occur. Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088.

What other drugs will affect eteplirsen?

Other drugs may affect eteplirsen, including prescription and over-the-counter medicines, vitamins, and herbal products. Tell each of your child's healthcare providers about all medicines the child uses now, and any medicine the child starts or stops using.

Where can I get more information?

Your pharmacist can provide more information about eteplirsen.