delandistrogene moxeparvovec-rokl 1.33x10exp13 vg/mL intravenous susp

Generic name:
Delandistrogene moxeparvovec - injection

Brand name(s)
Elevidys

This medication is used to treat a certain muscle disorder that can lead to muscle weakness (Duchenne muscular dystrophy). Delandistrogene moxeparvovec is made from a certain virus that has been specially prepared. The virus is used to carry a certain human gene into cells. This type of treatment is known as gene therapy.

This medication is given by slow injection into a vein as directed by the doctor. It is given as a single dose. The dosage is based on your child's body weight.

To reduce the risk of side effects, the doctor may also direct your child to take another medication by mouth (a corticosteroid such as prednisone) before and after your child receives delandistrogene moxeparvovec. Follow your doctor's instructions carefully.

Infusion reactions may happen during the infusion of this drug and for up to several hours afterward. Tell the doctor right away if your child has any symptoms of infusion reactions such as fast heartbeat, fast breathing, flushing, nausea, vomiting, fever, or chills. The doctor may give your child other medications to help with these side effects or may slow down or stop the treatment for some time.

Small amounts of virus may shed from the child's stool/urine for up to 1 month after the injection. Ask the doctor how to handle and dispose of waste materials (such as diapers) for up to 1 month after the injection.

See also How to Use section.

Remember that this medication has been prescribed because the doctor has judged that the benefit to your child is greater than the risk of side effects. Many people using this medication do not have serious side effects.

Tell the doctor right away if your child has any serious side effects, including:

• signs of liver disease (such as nausea/vomiting that doesn't stop, loss of appetite, stomach/abdominal pain, yellowing eyes/skin, dark urine)
• easy bleeding/bruising
• unexplained muscle aches/weakness
• difficulty swallowing/speaking

Get medical help right away if your child has any very serious side effects, including:

• chest pain
• shortness of breath

A very serious allergic reaction to this drug is rare. However, get medical help right away if you notice any symptoms of a serious allergic reaction, including:

• rash
• itching/swelling (especially of the face/tongue/throat)
• severe dizziness
• trouble breathing

This is not a complete list of possible side effects. If you notice other effects not listed above, contact your doctor or pharmacist.

In the US -

Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088 or at www.fda.gov/medwatch.

In Canada - Call your doctor for medical advice about side effects. You may report side effects to Health Canada at 1-866-234-2345.

Before this medication is given, tell the doctor or pharmacist if your child has any allergies. This product may contain inactive ingredients, which can cause allergic reactions or other problems. Talk to the pharmacist for more details.

Before using this medication, tell the doctor or pharmacist your child's medical history, especially of:

• liver problems (including cirrhosis, fatty liver disease, hepatitis B or C)
• current/recent infection
• heart problems

Delandistrogene moxeparvovec may make current infections worse. Stay away from anyone who has an infection that may easily spread (such as chickenpox, COVID-19, measles, flu). Talk to the doctor if your child has been exposed to an infection or for more details.

Tell the health care professional that your child has received this medication before having any immunizations/vaccinations. Avoid contact with people who have recently received live vaccines (such as flu vaccine inhaled through the nose).

Before having surgery, tell the doctor or dentist about all the products your child uses (including prescription drugs, nonprescription drugs, and herbal products).

This medication is not usually used by adults. It is unlikely to be used during pregnancy or breastfeeding. Consult your doctor if you have any questions about this medication.

Drug interactions may change how your child's medications work or increase the risk for serious side effects. This document does not contain all possible drug interactions. Keep a list of all the products you use (including prescription/nonprescription drugs and herbal products) and share it with the doctor and pharmacist. Do not start, stop, or change the dosage of any medicines without the doctor's approval.

If someone has overdosed and has serious symptoms such as passing out or trouble breathing, call 911. Otherwise, call a poison control center right away. US residents can call 1-800-222-1222. Canada residents can call 1-844-764-7669.

Lab and/or medical tests (such as liver function, complete blood count, troponin levels) must be done before your child starts receiving this medication and after your child receives it. Keep all medical and lab appointments.

After receiving this treatment, your child cannot donate blood, organs, tissues, or cells.

Not applicable.

Not applicable. This type of product is given in a hospital or clinic and will not be stored at home.

Your condition can cause complications in a medical emergency. For information about enrolling in MedicAlert, call 1-888-633-4298 (US) or 1-800-668-1507 (Canada).

HOW TO USE THIS INFORMATION: This is a summary and does NOT have all possible information about this product. This information does not assure that this product is safe, effective, or appropriate for you. This information is not individual medical advice and does not substitute for the advice of your health care professional. Always ask your health care professional for complete information about this product and your specific health needs.