Reviews Overview: Elevidys is a one-time gene therapy that treats Duchenne muscular dystrophy (DMD) in certain people. It works by giving the body a gene to make a shorter version of a protein that protects muscles. Common side effects include vomiting, nausea, fever, and lab test changes. Elevidys cause serious risks like liver injury, heart problems, infections, and reactions during the infusion, so careful monitoring is needed. Contact a health care provider right away if you notice symptoms such as yellowing of the skin or eyes, signs of infection, chest pain, trouble breathing, allergic reactions, or worsening muscle weakness.
Uses
What is Elevidys used for?
Elevidys (delandistrogene moxeparvovec-rokl) is commonly used to treat Duchenne muscular dystrophy (DMD). DMD is a progressive, genetic disease that affects muscles, causing them to become weaker over time.
Learn more about Elevidys for Duchenne muscular dystrophy (DMD).
Elevidys may also be used for other conditions as determined by your health care provider.
How does Elevidys work (mechanism of action)?
In DMD, you have a change in your genes, which is called a genetic mutation. This change causes the dystrophin protein to not work correctly. Your muscles need dystrophin to stay healthy and be strong. With less dystrophin, muscles become weak over time.
Elevidys delivers a gene that signals your body to make a shorter version of dystrophin. This can help protect muscles from damage and keep muscles stronger for a longer time.
How is Elevidys supplied (dosage forms)?
Elevidys is available in an injectable form that is given by a health care provider.
How should I store Elevidys?
Elevidys is 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 Elevidys?
The most common side effects of Elevidys are listed below. Tell your health care provider if you have any of these side effects that bother you.
- Vomiting
- Nausea
- Fever
- Low platelet levels (see below)
- Liver problems (see below)
There may be other side effects of Elevidys 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 (800-332-1088). 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 Elevidys?
While less common, the most serious side effects of Elevidys are described below, along with what to do if they happen.
Severe Allergic Reactions. Elevidys may cause allergic reactions, which can be serious. Tell your health care provider right away if you are having 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 or skin rash
- Bumps on the skin called hives that can be red, pink, white, or brown depending on your skin tone
- Nausea or vomiting
- Dizziness, feeling lightheaded, or fainting
- Stomach cramps
- Joint pain
Liver Problems. Liver damage, also called hepatotoxicity, can happen when taking Elevidys and may be sudden and serious or even life-threatening. People with existing liver problems may have a higher risk. You will need to get frequent blood tests, called liver function tests (LFTs) to check the health of your liver before and after Elevidys treatment. Call your health care provider right away if you have any of the following symptoms of liver damage.
- Nausea or vomiting
- Stomach or belly pain
- Fever
- Weakness or unusual tiredness
- Itching
- Loss of appetite
- Light-colored poop
- Dark-colored urine (pee)
- Your skin or the whites of your eyes turning yellowish in color (also called jaundice)
Infusion Reactions. Serious reactions can happen while you are receiving Elevidys or within several hours after you receive the infusion. Your health care provider may give you medicines before you receive Elevidys to help prevent infusion reactions. Your infusion may need to be slowed or stopped if you have a reaction during treatment. Tell your health care provider right away if you have any of the following symptoms during or after your infusion.
- Fast heart rate
- Trouble breathing or fast breathing
- Itchy or swollen lips
- Redness of your face and neck
- Nausea or vomiting
- Fever or chills
- Widening nostrils
Inflamed Muscles. Some conditions and medicines, like Elevidys, can cause myositis, which is inflammation in muscles. When using new gene medicines, the immune system may ramp up and cause more inflammation as it tries to “fight off” the new medicine. When this inflammation affects the muscles, you may experience more weakness, swelling, and pain. Contact your health care provider right away if you have any of the following symptoms of myositis.
- Increased muscle weakness
- Muscle pain or tenderness
- Trouble swallowing (dysphagia) or talking
- Trouble breathing or shortness of breath (dyspnea)
Inflammation of the Heart Muscle. Myocarditis is when your heart muscle becomes inflamed or irritated. This can affect how well your heart pumps blood. Elevidys can cause the immune system to produce more inflammation around the heart muscle. Your health care provider may do blood tests to check for damage to the heart muscle. Contact your health care right away if you have the following symptoms of heart damage.
- Chest pain
- Shortness of breath or trouble breathing
Severely Low Platelet Levels. Elevidys can cause you to have a severely low number of platelets in your blood, also called thrombocytopenia. Platelets help to form blood clots. Having a low level of platelets may increase your risk of bleeding. Tell your health care provider if you have any of the following symptoms of low platelet levels.
- Easy bruising or bleeding
- Bleeding that does not stop
- Nosebleeds
Warnings & Precautions
Who should not use Elevidys?
Allergies to Ingredients. People who are allergic to any of the following should not use Elevidys.
- Elevidys
- Delandistrogene moxeparvovec-rokl
- Any of the ingredients in the specific product dispensed
Your health care provider can tell you all the ingredients in Elevidys.
Genetic Variations. People with DMD can have different variations in the DMD gene, also called mutations. Elevidys should not be used if you have specific genetic mutations. Your health care provider may test you for these mutations before using Elevidys.
Antibodies. Your immune system uses antibodies to protect you from illness. Elevidys uses a special tool, called a vector, to deliver a gene for the dystrophin protein to your muscle cells. Some people may have antibodies to this vector. If they have too many antibodies to the vector, it may stop Elevidys from working correctly or cause serious side effects.
What should I know about Elevidys before using it?
Elevidys is usually given by a health care provider in a hospital or clinic. Your health care provider will store and give you the medicine.
Your health care provider may tell you to take certain medicines before or after you receive Elevidys. These medicines can help to prevent certain side effects.
Treatment with Elevidys involves careful planning before, during, and after the infusion to help keep you as safe as possible. It is very important to keep all appointments with your health care team. You will need to get blood tests before, during, or after the Elevidys infusion. This is because Elevidys can cause serious side effects, including liver problems, and requires close monitoring before and after treatment. It is recommended to stay near a health care facility for at least 2 months after treatment.
After receiving Elevidys, small amounts of the vector (an inactive virus used to carry the gene therapy) can be found in bodily fluids and poop for a short time. This is normal and does not mean you have or are spreading an infection. As a precaution, practice good hand hygiene and place any soiled materials in a sealed bag before throwing them away. You can throw the sealed bag in the regular trash. These precautions are usually needed for about a month after the Elevidys infusion.
What should I tell my health care provider before using Elevidys?
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 know if Elevidys is right for you.
In particular, make sure that you discuss any of the following.
Infections. Tell your health care provider if you are being treated for any infection, have an infection that keeps coming back, or think you have an infection or have any of the following symptoms.
- Burning or stinging when you pee
- Cough or coughing up blood
- Fever, sweats, chills, or muscle aches
- Shortness of breath
- Diarrhea or stomach pain
- Weight loss
Vaccines. Tell your health care provider about your vaccine history or if you have any vaccines scheduled. If possible, your vaccines should be up-to-date before you start Elevidys. You should not receive any vaccines for at least 4 weeks before starting the corticosteroid medicine that is taken before the Elevidys infusion.
Current and Past Health Conditions. Tell your health care provider if you have any of the following.
- Liver problems, such as increased liver enzymes or hepatitis
- Heart disease
Pregnancy. Elevidys should not be used in pregnant women. Tell your health care provider if you are or plan to become pregnant.
Breastfeeding. It is not known if Elevidys passes into breast milk. Tell your health care provider if you are breastfeeding or plan to breastfeed.
How can I get more information about Elevidys?
For more information about Elevidys, you can visit the drugmaker's website at www.elevidys.com or call them at 888-727-3782.
Interactions
Does Elevidys interact with foods or drinks?
There are no known interactions between Elevidys and foods or drinks.
It is unknown if drinking alcohol will affect Elevidys.
Does Elevidys interact with other medicines (drug interactions)?
Tell your health care provider about your vaccination history. If a vaccine is needed, your health care provider will tell you when to get the vaccine before receiving the Elevidys infusion.
This may not be a complete list of medicines that can interact with Elevidys. Always check with your health care provider.
Overdose/Missed Dose
What should I do if I accidentally use too much Elevidys?
If you or someone else has used too much Elevidys, 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 Elevidys?
Elevidys is a single, one-time treatment, that is given by a health care provider at a hospital or clinic.
Add WebMD as a Preferred Source to see more of our trusted content across Google Search, AI Overviews, and AI Mode