Patient information: uses, side effects, how to take - Elevidys
What is ELEVIDYS?
ELEVIDYS is a prescription gene therapy used to treat ambulatory individuals at least 4 years old with Duchenne muscular dystrophy (DMD) who have a confirmed mutation in the DMD gene.
ELEVIDYS is not recommended for individuals with:
- Preexisting liver problems or liver infection because of the high risk of rapid serious liver injury and rapid liver failure
- Recent vaccination (within 4 weeks of ELEVIDYS treatment)
- Current or recent infections (within 4 weeks of ELEVIDYS treatment)
Who should not take ELEVIDYS?
You/Your child should not receive ELEVIDYS if they have a certain type of genetic mutation, called a deletion, involving any portion of or the entire exon 8 and/or exon 9 in the DMD gene.
Before taking ELEVIDYS, tell your child's doctor about all your child's medical conditions, including if they have:
- preexisting liver problems
- current or recent infection
- recent vaccinations (within 4 weeks)
How should your child receive ELEVIDYS?
Before receiving ELEVIDYS, you/your child will need to get blood tests to check:
- the amount of antibodies to ELEVIDYS
- liver function
- platelets, which is a kind of blood cell that helps you stop bleeding
- heart protein
- Your/Your child's healthcare provider will give ELEVIDYS into a vein of the arm through an intravenous (IV) line that may last around 1 to 2 hours or longer.
- Your/Your child's healthcare provider will check on you/your child during and at least 3 hours after the infusion for infusion-related reactions. See “What is the most important information I should know about ELEVIDYS?” If an infusion-related reaction occurs during their ELEVIDYS infusion, your/your child's healthcare provider may decide to give ELEVIDYS more slowly or stop the infusion.
What are the possible side effects of ELEVIDYS?
- ELEVIDYS can cause serious side effects. See “What is the most important information I should know about ELEVIDYS?”
The most common side effects in individuals treated with ELEVIDYS include:
- vomiting
- nausea
- liver injury
- fever
- lower number of platelets, which is a kind of blood cell that helps you stop bleeding
- higher levels of heart protein that can detect damage to muscle cells in the heart (troponin-I)
Tell your/your child's doctor about any side effects that bother you/your child or that do not go away. These are not all the possible side effects of ELEVIDYS.
Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088.
You may also report side effects to Sarepta Therapeutics, Inc. at 1-888-727-3782.
What should you/your child avoid after receiving ELEVIDYS?
Avoid taking new medications that are known to damage the liver as this may make liver problems worse. Tell your doctor about all the medicines you/your child takes, including prescription and over-the counter medicines, vitamins, and herbal supplements.
- Discuss with your/your child's doctor before receiving a vaccine after ELEVIDYS treatment.
- Avoid missing any dose of corticosteroids. Contact your/your child's doctor immediately if this happens.
See “What is the most important information I should know about ELEVIDYS?”
General information about the safe and effective use of ELEVIDYS.
Medicines are sometimes prescribed for purposes other than those listed in a Medication Guide. You can ask your/your child's pharmacist or doctor for information about ELEVIDYS that is written for health professionals.
What are the ingredients in ELEVIDYS?
Active ingredients: delandistrogene moxeparvovec-rokl
Inactive ingredients: sodium chloride, tromethamine hydrochloride, tromethamine, magnesium chloride, poloxamer 188
Manufactured for: Sarepta Therapeutics, Inc. Cambridge, MA 02142 USA U.S. license number 2308
SAREPTA, SAREPTA THERAPEUTICS and ELEVIDYS are trademarks of Sarepta Therapeutics, Inc.
©2025 Sarepta Therapeutics, Inc.