Description and Brand Names

Drug information provided by: Merative, Micromedex®

US Brand Name

  1. Elevidys

Descriptions


Delandistrogene moxeparvovec-rokl injection is used to treat Duchenne muscular dystrophy (DMD) in patients with a confirmed specific genetic mutation. DMD is a rare, inherited muscle disease that is caused by absence of a protein called dystrophin, which helps keep muscle cells intact.

This medicine is to be given only by or under the direct supervision of a doctor.

This product is available in the following dosage forms:

  • Suspension

Before Using

In deciding to use a medicine, the risks of taking the medicine must be weighed against the good it will do. This is a decision you and your doctor will make. For this medicine, the following should be considered:

Allergies

Tell your doctor if you have ever had any unusual or allergic reaction to this medicine or any other medicines. Also tell your health care professional if you have any other types of allergies, such as to foods, dyes, preservatives, or animals. For non-prescription products, read the label or package ingredients carefully.

Pediatric

Appropriate studies have not been performed on the relationship of age to the effects of delandistrogene moxeparvovec-rokl injection in children younger than 3 years of age and children 6 years of age and older. Safety and efficacy have not been established.

Geriatric

No information is available on the relationship of age to the effects of delandistrogene moxeparvovec-rokl injection in geriatric patients.

Breastfeeding

There are no adequate studies in women for determining infant risk when using this medication during breastfeeding. Weigh the potential benefits against the potential risks before taking this medication while breastfeeding.

Drug Interactions

Although certain medicines should not be used together at all, in other cases two different medicines may be used together even if an interaction might occur. In these cases, your doctor may want to change the dose, or other precautions may be necessary. Tell your healthcare professional if you are taking any other prescription or nonprescription (over-the-counter [OTC]) medicine.

Other Interactions

Certain medicines should not be used at or around the time of eating food or eating certain types of food since interactions may occur. Using alcohol or tobacco with certain medicines may also cause interactions to occur. Discuss with your healthcare professional the use of your medicine with food, alcohol, or tobacco.

Other Medical Problems

The presence of other medical problems may affect the use of this medicine. Make sure you tell your doctor if you have any other medical problems, especially:

  • Heart disease or
  • Liver disease or
  • Muscle problems—Use with caution. May make these conditions worse.
  • Infection—Should be treated first before starting this medicine.
  • Patients with deleted exon 8 and/or 9 in DMD gene or
  • Patients with elevated anti-AAVrh74 antibody—Use is not recommended in patients with these conditions.

Proper Use

A nurse or other trained health professional will give you or your child this medicine in a medical facility. This medicine is given through a needle placed in one of your veins. It must be given slowly, so the needle should remain in place for 1 to 2 hours.

Your doctor will also give you other medicines (eg, steroid medicine) before and after receiving this medicine.

Precautions

Your doctor will check your child's progress closely while you are receiving this medicine to see if it is working properly and to decide if you or your child should continue to receive it. Blood and urine tests may be needed to check for unwanted effects.

Check with your doctor right away if your child has pain or tenderness in the upper stomach, pale stools, dark urine, loss of appetite, nausea, unusual tiredness or weakness, or yellow eyes or skin. These could be symptoms of a serious liver problem.

This medicine may cause muscle problems (eg, myositis). Check with your doctor right away if your child has difficulty swallowing, muscle pain or weakness, trouble breathing, or unusual tiredness or weakness.

Check with your doctor right away if your child has chest pain or discomfort, fever and chills, fast heartbeat, or trouble breathing. These maybe symptoms of myocarditis (swelling of the heart).

Your child’s vaccines should be completed at least 4 weeks before starting steroid medicine. Ask your child’s doctor if you have any questions about this.

Side Effects

Along with its needed effects, a medicine may cause some unwanted effects. Although not all of these side effects may occur, if they do occur they may need medical attention.

Check with your doctor or nurse immediately if any of the following side effects occur:

More common

  1. Black, tarry stools
  2. bleeding gums
  3. blood in the urine or stools
  4. pinpoint red spots on the skin
  5. unusual bleeding or bruising

Incidence not known

  1. Chest pain or discomfort
  2. cough
  3. dark urine
  4. fast heartbeat
  5. fever and chills
  6. loss of appetite
  7. muscle pain
  8. nausea or vomiting
  9. redness or swelling in the ear
  10. sore throat
  11. stomach pain, severe
  12. stuffy or runny nose
  13. trouble breathing
  14. unusual tiredness or weakness
  15. yellow eyes or skin

Other side effects not listed may also occur in some patients. If you notice any other effects, check with your healthcare professional.

Call your doctor for medical advice about side effects. You may report side effects to the FDA at 1-800-FDA-1088.

Portions of this document last updated: Feb. 01, 2024

Original article: https://www.mayoclinic.org/es/drugs-supplements/delandistrogene-moxeparvovec-rokl-intravenous-route/description/drg-20554229