4 mins read

Overview: Exondys 51 is used to treat Duchenne muscular dystrophy (DMD) in people who have a specific mutation in the DMD gene. This medicine helps these people to make a shorter but working protein called dystrophin that is needed for muscles to work. Common side effects include feeling off balance or vomiting. Your health care provider will give you this medicine by an injection into your vein (IV).

Uses

What is Exondys 51 used for?

Exondys 51 (eteplirsen) is commonly used to treat Duchenne muscular dystrophy (DMD) in certain people.

Exondys 51 may also be used for other conditions as determined by your health care provider.

How does Exondys 51 work (mechanism of action)?

Dystrophin is a protein that is needed for muscles to work. Some people with Duchenne muscular dystrophy have a specific change in the DMD gene that can be treated with a process called exon 51 skipping. Exondys 51 is a medicine that helps these people to make a shorter but working dystrophin protein. 

How is Exondys 51 supplied (dosage forms)?

Exondys 51 is available in an injectable form that is given by a health care provider.

How should I store Exondys 51?

Exondys 51 is usually given by a health care provider in a hospital or clinic. You will not store it at home.

Side Effects

What are the most common side effects of Exondys 51?

The most common side effects of Exondys 51 are listed below. Tell your health care provider if you have any of these side effects that bother you.

  • Feeling off balance
  • Vomiting

There may be other side effects of Exondys 51 that are not listed here. Contact your health care provider if you think you are having a side effect of a medicine. In the U.S., you can report side effects to the FDA at www.fda.gov/medwatch or by calling 800-FDA-1088. In Canada, you can report side effects to Health Canada at www.health.gc.ca/medeffect or by calling 866-234-2345.

What are the serious side effects of Exondys 51?

While less common, the most serious side effects of Exondys 51 are described below, along with what to do if they happen.

Severe Allergic Reactions.Exondys 51 may cause allergic reactions, which can be serious. Get help right away if you have any of the following symptoms of a serious allergic reaction.

  • Breathing problems or wheezing
  • Racing heart
  • Fever or general ill feeling
  • Swollen lymph nodes
  • Swelling of the face, lips, mouth, tongue, or throat
  • Trouble swallowing or throat tightness
  • Itching, skin rash, or pale red bumps on the skin called hives
  • Nausea or vomiting
  • Dizziness, feeling lightheaded, or fainting
  • Stomach cramps
  • Joint pain

Warnings & Precautions

Who should not use Exondys 51?

Allergies to Ingredients. People who are allergic to any of the following should not use Exondys 51.

  • Exondys 51 
  • Eteplirsen
  • Any of the ingredients in the specific product dispensed

Your pharmacist can tell you all of the ingredients in Exondys 51.

What should I know about Exondys 51 before using it?

Exondys 51 will be injected into your vein (intravenous) by a health care provider in a hospital or clinic.

What should I tell my health care provider before using Exondys 51?

Tell your health care provider about all of your health conditions and any prescription or over-the-counter (OTC) medicines, vitamins/minerals, herbal products, and other supplements you are using. This will help them determine if Exondys 51 is right for you.

In particular, make sure that you discuss any of the following.

Pregnancy. It is not known if or how Exondys 51 could affect pregnancy or harm an unborn baby. Tell your health care provider if you are pregnant or plan to become pregnant.

Breastfeeding. It is not known if Exondys 51 passes into breast milk. Tell your health care provider if you are breastfeeding or plan to breastfeed.

How can I get more information about Exondys 51?

For more information about Exondys 51, you can visit the drugmaker’s website at www.exondys51.com or call them at 888-727-3782.

Interactions

Does Exondys 51 interact with foods or drinks?

There are no known interactions between Exondys 51 and foods or drinks.

It is unknown if drinking alcohol will affect Exondys 51. 

Does Exondys 51 interact with other medicines (drug interactions)?

There are no known interactions between Exondys 51 and other medicines. Always tell your health care provider about any prescription or over-the-counter (OTC) medicines, vitamins/minerals, herbal products, and other supplements you are using.

Does interact with other drugs you are taking?

Enter your medication into the WebMD interaction checker

Overdose/Missed Dose

What should I do if I accidentally use too much Exondys 51?

If you or someone else has used too much Exondys 51, get medical help right away, call 911, or contact a Poison Control center at 800-222-1222.

What should I do if I miss a dose of Exondys 51?

If you miss an appointment to get your Exondys 51 dose, call to make another appointment as soon as possible.