Delandistrogene moxeparvovec

Generic name: delandistrogene moxeparvovec
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Dosage form: intravenous kit (rokl)
Drug class: Miscellaneous uncategorized agents

Medically reviewed by Drugs.com on Dec 14, 2023. Written by Cerner Multum.

What is delandistrogene moxeparvovec?

Delandistrogene moxeparvovec is used to treat Duchenne muscular dystrophy in children who are 4 to 5 years old that have a specific gene mutation.

The doctor will make sure your child has the correct gene mutation to be treated with delandistrogene moxeparvovec.

Delandistrogene moxeparvovec may also be used for purposes not listed in this medication guide.

Delandistrogene moxeparvovec side effects

Get emergency medical help if you have signs of an allergic reaction: hives, difficult breathing, swelling of your face, lips, tongue, or throat.

Delandistrogene moxeparvovec can cause serious side effects (even weeks or months after your child received this treatment). Tell your child's doctor right away if they have:

• liver problems--loss of appetite, nausea, vomiting, stomach pain (upper right side), tiredness, itching, dark urine, clay-colored stools, jaundice (yellowing of the skin or eyes);

• cold symptoms--runny nose, sneezing, sore throat, fever, cough;

• chest pain, shortness of breath; or

• trouble speaking or swallowing, increased muscle pain, weakness or tenderness.

Common side effects of delandistrogene moxeparvovec may include:

• fever;

• low levels of platelets in your blood;

• vomiting, nausea; or

• abnormal liver function tests.

This is not a complete list of side effects and others may occur. Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088.

Related/similar drugs

Warnings

Delandistrogene moxeparvovec can cause serious side effects (even weeks or months after your child received this treatment). Tell your child's doctor if they have: chest pain, shortness of breath, trouble speaking or swallowing, increased muscle pain, weakness, tenderness, jaundice (yellowing of the skin or eyes), or cold symptoms.

Before taking this medicine

Your child should not receive delandistrogene moxeparvovec if they have a certain type of gene mutation. Ask your child's doctor about this risk.

Tell your child's doctor if they have or have ever had:

• recently had or have an infection;

• a history of high levels of a specific type of antibody called anti-AAVrh74;

• heart problems; or

• liver disease.

Make sure your child is current on all vaccines at least 4 weeks before starting delandistrogene moxeparvovec.

How is delandistrogene moxeparvovec given?

Your child's doctor will perform tests before starting treatment with delandistrogene moxeparvovec to make sure it is the right treatment for your child.

Doses are based on weight. Your doctor will confirm your child's weight before starting treatment with delandistrogene moxeparvovec.

Delandistrogene moxeparvovec is given in one dose and injected into a vein with an infusion pump. A healthcare provider will give this injection.

Your child will need frequent blood tests before and after receiving delandistrogene moxeparvovec.

Your child will be given other medications by mouth before and after receiving delandistrogene moxeparvovec to help prevent serious side effects. Your child must keep taking these medicines for as long as the doctor has prescribed. Tell the doctor right away if your child misses a dose or vomits shortly after taking these medications.

The child's caregiver should practice proper hand hygiene when coming into direct contact with the child's body waste for 1 month after the child receives delandistrogene moxeparvovec. Place any materials that may have your child's body waste in a sealed plastic bag and dispose in regular trash.

What happens if I miss a dose?

Delandistrogene moxeparvovec is given as a single dose and does not have a dosing schedule.

What happens if I overdose?

In a medical setting an overdose would be treated quickly.

What should I avoid before receiving delandistrogene moxeparvovec?

Follow your child's doctor's instructions about any restrictions on food, beverages, or activity.

What other drugs will affect delandistrogene moxeparvovec?

Other drugs may affect delandistrogene moxeparvovec, including prescription and over-the-counter medicines, vitamins, and herbal products. Tell your doctor about all other medicines you use.

Further information

Remember, keep this and all other medicines out of the reach of children, never share your medicines with others, and use this medication only for the indication prescribed.

Always consult your healthcare provider to ensure the information displayed on this page applies to your personal circumstances.

Medical Disclaimer

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