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Exondys 51

Generic Name: eteplirsen (e TEP lir sen)
Brand Name: Exondys 51

What is Exondys 51 (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 is the most important information I should know about Exondys 51 (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 should I discuss with my healthcare provider before receiving Exondys 51 (eteplirsen)?

Tell your child's doctor if any prior eteplirsen injections caused a severe allergic reaction in your child.

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 Exondys 51 (eteplirsen)given?

Eteplirsen is injected into a vein through an IV. A healthcare provider will give your child 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 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?

Call your child's doctor for instructions if you miss an appointment for your child's eteplirsen injection.

What happens if I overdose?

Seek emergency medical attention or call the Poison Help line at 1-800-222-1222.

What should I avoid while receiving Exondys 51 (eteplirsen)?

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

Exondys 51 (eteplirsen) side effects

Get emergency medical help if your child has signs of an allergic reaction: hives; difficult breathing; swelling of your face, lips, tongue, or throat.

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.

See also: Side effects (in more detail)

What other drugs will affect Exondys 51 (eteplirsen)?

Other drugs may interact with 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.
  • Remember, keep this and all other medicines out of the reach of children, never share your medicines with others, and use this medication only for the indication prescribed.
  • Disclaimer: Every effort has been made to ensure that the information provided by Cerner Multum, Inc. ('Multum') is accurate, up-to-date, and complete, but no guarantee is made to that effect. Drug information contained herein may be time sensitive. Multum information has been compiled for use by healthcare practitioners and consumers in the United States and therefore Multum does not warrant that uses outside of the United States are appropriate, unless specifically indicated otherwise. Multum's drug information does not endorse drugs, diagnose patients or recommend therapy. Multum's drug information is an informational resource designed to assist licensed healthcare practitioners in caring for their patients and/or to serve consumers viewing this service as a supplement to, and not a substitute for, the expertise, skill, knowledge and judgment of healthcare practitioners. The absence of a warning for a given drug or drug combination in no way should be construed to indicate that the drug or drug combination is safe, effective or appropriate for any given patient. Multum does not assume any responsibility for any aspect of healthcare administered with the aid of information Multum provides. The information contained herein is not intended to cover all possible uses, directions, precautions, warnings, drug interactions, allergic reactions, or adverse effects. If you have questions about the drugs you are taking, check with your doctor, nurse or pharmacist.

Copyright 1996-2012 Cerner Multum, Inc. Version: 1.01.

Date modified: November 30, 2016
Last reviewed: October 12, 2016

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