Elevidys (delandistrogene moxeparvovec) is a prescription gene therapy that’s used to treat Duchenne muscular dystrophy. Elevidys is a single-dose treatment that’s given as an intravenous infusion.

To learn more about Elevidys’s uses, see the “What is Elevidys used for?” section below.

Elevidys basics

Elevidys contains the active ingredient delandistrogene moxeparvovec-rokl.

Elevidys is a biologic medication. A biologic is made from parts of living organisms. It’s available only as a brand-name drug. It isn’t available in a biosimilar version.

Biosimilars are like generic drugs. But unlike generics, which are made for non-biologic drugs, biosimilars are made for biologic drugs.

Biosimilars will have the same active ingredient core name as the brand-name drug. But they’ll have unique suffixes. This is why “-rokl” appears at the end of the name of the active ingredient. This is to show that it’s distinct from biosimilars that may be created in the future.

Elevidys is used to treat a rare genetic condition called Duchenne muscular dystrophy (DMD).

Specifically, the FDA has approved Elevidys to treat DMD in people ages 4 years and older who have a confirmed mutation (abnormal change) in the DMD gene and:

  • are ambulatory, which means the person can still walk without assistance, or
  • are non-ambulatory,* which means the person is no longer able to walk

DMD occurs when an inherited DMD gene is abnormal or has missing parts. This mutation causes your body to make little to no dystrophin, which is a protein that helps muscle cells work properly and protects muscles from getting damaged.

Symptoms of DMD usually start before age 5 and may include muscle weakness, muscle wasting, trouble walking, and learning disabilities. Over time, serious heart and breathing problems may slowly develop.

Elevidys is a type of gene therapy. It provides your body with a copy of a gene that codes for a shortened version of the dystrophin protein called micro-dystrophin. After receiving Elevidys treatment, your body starts making micro-dystrophin, which may help improve muscle function. In studies, some ambulatory children were able to get up from the floor or walk faster after receiving treatment with Elevidys compared to children who received a placebo.

* For this purpose, Elevidys has received accelerated approval from the FDA. This means that Elevidys was approved based on early studies showing that the micro-dystrophin protein was present in the muscles of non-ambulatory children who received Elevidys. The FDA’s decision to fully approve Elevidys will be made after more clinical studies confirm its benefits.

Like most drugs, Elevidys may cause mild to serious side effects. The lists below contain some of the more common side effects Elevidys may cause, but they don’t include all possible side effects.

Keep in mind that side effects of a drug can depend on:

  • your age
  • other health conditions you have
  • other medications you take

Your doctor or pharmacist can tell you more about the potential side effects of Elevidys. They can also suggest ways to help reduce side effects.

Mild side effects

Here’s a list of some of the mild side effects that Elevidys can cause. To learn about other mild side effects, talk with your doctor or pharmacist, or read Elevidys’s prescribing information.

Mild side effects of Elevidys that have been reported include:

  • nausea
  • vomiting
  • fever

Mild side effects of many drugs may go away within a few days to a couple of weeks. But if they become bothersome, talk with your doctor or pharmacist.

Serious side effects

Serious side effects from Elevidys can occur, but they aren’t common. If you have serious side effects from Elevidys, call your doctor right away. But if you think you’re having a medical emergency, you should call 911 or your local emergency number.

Serious side effects of Elevidys that have been reported include:

  • infusion reactions (severe reactions during or after an infusion), such as rapid heart rate, breathing problems, lip swelling, or rash
  • acute (sudden or short-term) liver injury
  • elevated liver enzymes, which may be a sign of liver injury
  • acute myocarditis (inflammation of heart muscle)
  • thrombocytopenia (low platelet count)
Allergic reaction

For some people, Elevidys can cause an allergic reaction. This side effect occurred in the drug’s studies.

In general, symptoms of allergic reaction can be mild or serious.

What might help

If you have mild symptoms of an allergic reaction, such as a mild rash, call your doctor right away. They may suggest a treatment to manage your symptoms and can determine whether you should keep taking Elevidys.

If you have symptoms of a severe allergic reaction, such as swelling or trouble breathing, call 911 or your local emergency number right away. These symptoms could be life threatening and require immediate medical care.

If your doctor confirms you’ve had a serious allergic reaction to Elevidys, they may have you switch to a different treatment.

Your doctor will recommend the dosage of Elevidys that’s right for you.

Form and strength

Elevidys is available as a liquid suspension that’s given as an intravenous (IV) infusion (an injection into your vein over a period of time).

Elevidys comes in a customized kit that includes multiple vials of the drug. Each vial contains 10 milliliters (mL) of the drug. A healthcare professional will use the kit to prepare a suspension that has a concentration of 1.33 × 1014 vector genomes per milliliter (vg/mL). (Each “vector genome” refers to a copy of a virus-like vehicle that carries genetic material.)

The number of vials in each kit will be dependent on your weight to ensure the correct dosage matches your specific needs.

Recommended dosage

Your doctor may determine your dose of Elevidys based on your body weight in kilograms (kg).

For people who weigh less than 70 kg, the recommended Elevidys dose is 1.33 × 1014 vector genomes (vg) per kg of body weight.

For people who weigh 70 kg or more, the recommended Elevidys dose is a fixed dose of 9.31 × 1015 vector genomes (vg).

How Elevidys is given

Elevidys is a single-dose treatment. It’s given as an IV infusion. You’ll receive the infusion at a hospital or treatment center. The infusion usually takes 1 to 2 hours.

Your doctor will explain how and when Elevidys will be given to you. They’ll also explain how to prepare for the infusion, including details about medications to take before Elevidys treatment. In addition, they’ll explain what to expect and provide details about medications to take after receiving Elevidys.

You can learn more about what to expect with Elevidys in the drugmaker’s treatment guide.

Questions about using Elevidys

Below are some common questions about using Elevidys.

  • Will I need to use Elevidys long term? Elevidys is a one-dose treatment. After a single infusion, the drug is thought to work long-term to treat DMD in certain people.
  • How long does Elevidys take to work? Elevidys begins to work shortly after you receive the infusion. But you likely won’t feel the drug working in your body. Your doctor may order tests and do physical exams to check how well Elevidys is working to treat your condition.

Below is important information you should consider before using Elevidys.

Interactions

Taking a drug with certain medications, vaccines, foods, and other things can affect how the drug works. These effects are called interactions.

It isn’t clear if Elevidys itself has interactions. However, before Elevidys is given, you’ll need to receive treatment with corticosteroids. Corticosteroids can lower the activity of your immune system and may interact with vaccines. Because of this, your doctor will likely advise getting any routine or recommended vaccines at least 4 weeks before starting corticosteroids in preparation for Elevidys treatment.

Before taking Elevidys, talk with your doctor and pharmacist. Tell them about all prescription, over-the-counter, and other drugs you take. Also tell them about any vitamins, herbs, and supplements you use. Sharing this information can help you avoid potential interactions.

If you have questions about drug interactions that may affect you, talk with your doctor or pharmacist.

Elevidys and alcohol

Alcohol is not known to interact with Elevidys.

Talk with your doctor to determine whether it’s safe for you to consume alcohol after receiving Elevidys treatment.

Warnings

Elevidys can sometimes cause harmful effects in people who have certain conditions. This is known as a drug-condition interaction. Other factors may also affect whether Elevidys is a good treatment option for you.

Talk with your doctor about your health history before you take Elevidys. Be sure to tell them if any of the following factors apply to you:

  • a DMD gene mutation (abnormal change) known as a deletion in exon 8 or exon 9
  • higher levels of certain antibodies (immune system proteins) that recognize adeno-associated viruses*
  • active infection
  • liver problems
  • previous allergic reaction to Elevidys

* Having above a certain level of these antibodies in your body could prevent Elevidys from working effectively. Your doctor can tell you more about this.

Whether you have health insurance or not, cost may be a factor when you’re considering Elevidys. What you’ll pay for Elevidys may depend on several things, such as your treatment plan and the pharmacy you use.

If you have questions about how to pay for Elevidys, talk with your doctor or pharmacist. A program called SareptAssist may also be available.

You can also check out this article to learn more about saving money on prescriptions.

Currently, Elevidys is the only gene therapy available for DMD. Other drugs are available that can treat this condition. If you’d like to explore an alternative to Elevidys, talk with your doctor. They can tell you about other medications that might work for you.

Examples of other drugs that may be used to treat DMD are:

Disclaimer: Healthline has made every effort to make certain that all information is factually correct, comprehensive, and up to date. However, this article should not be used as a substitute for the knowledge and expertise of a licensed healthcare professional. You should always consult your doctor or another healthcare professional before taking any medication. The drug information contained herein is subject to change and is not intended to cover all possible uses, directions, precautions, warnings, drug interactions, allergic reactions, or adverse effects. The absence of warnings or other information for a given drug does not indicate that the drug or drug combination is safe, effective, or appropriate for all patients or all specific uses.