Why is this medicine prescribed?
Delandistrogene moxeparvovec-rokl injection is used to treat Duchenne muscular dystrophy (DMD; a progressive disease in which the muscles do not function properly) in certain children 4 through 5 years of age. It is in a class of medications called gene therapy. It works by replacing a defective gene in muscles which helps improve muscle function.
Are there other uses for this medicine?
This medication may be prescribed for other uses; ask your doctor or pharmacist for more information.
How should this medicine be used?
Delandistrogene moxeparvovec-rokl comes as a suspension (liquid) to be injected intravenously (into a vein) by a doctor or nurse in a healthcare setting. It is usually infused slowly over at least 1 to 2 hours as a single dose.
Delandistrogene moxeparvovec-rokl may cause serious immune response reactions after your child receives the infusion. The doctor will prescribe a corticosteroid (a type of medication used to prevent swelling and inflammation) for your child to take 1–7 days before treatment with delandistrogene moxeparvovec-rokl injection and for at least 60 days after the infusion to help prevent these reactions. Call the doctor if your child misses a corticosteroid dose or vomits after taking a dose of corticosteroids.
Ask your pharmacist or doctor for a copy of the manufacturer's information for the patient.
What special precautions should I follow?
Before receiving delandistrogene moxeparvovec-rokl injection,
- tell the doctor and pharmacist if your child is allergic to delandistrogene moxeparvovec-rokl, any other medications, or any of the ingredients in delandistrogene moxeparvovec-rokl injection. Ask your pharmacist for a list of the ingredients.
- tell the doctor and pharmacist what other prescription and nonprescription medications, vitamins, nutritional supplements, and herbal products your child is taking or plan to take. The doctor may need to change the doses of your medications or monitor you carefully for side effects.
- tell the doctor if your child has an infection or if your child has or has ever had any heart problems; low platelet counts; or liver problems, including hepatitis B or C (a virus that infects the liver and may cause severe liver damage) or cirrhosis (scarring) of the liver.
- check with the doctor to see if your child needs to receive any vaccinations. It is important to have all vaccines appropriate for your child's age before beginning treatment with delandistrogene moxeparvovec-rokl injection. Also tell the doctor if your child has recently (within 4 weeks) received any vaccinations. Your child should not have any vaccinations before or after their treatment with delandistrogene moxeparvovec-rokl injection without talking to the doctor.
- you should know that delandistrogene moxeparvovec-rokl injection contains a virus that could spread and infect other people. You should maintain proper hygiene and wash your hands if you come in contact with your child's bodily fluids or waste (urine, feces, saliva). You should be sure to put all contaminated materials that may contain your child's bodily fluids or waste into a sealed plastic bag and throw them away into the garbage for 1 month after the infusion.
- if your child is having surgery, including dental surgery, tell the doctor or dentist that they have received delandistrogene moxeparvovec-rokl injection.
What special dietary instructions should I follow?
Unless your doctor tells you otherwise, continue your normal diet.
What side effects can this medicine cause?
Delandistrogene moxeparvovec-rokl injection may cause side effects. Tell the doctor if any of these symptoms are severe or do not go away:
Some side effects can be serious. If you experience any of these symptoms, call the doctor immediately or get emergency medical treatment:
- increased muscle pain, tenderness, or weakness
- difficulty breathing, swallowing, or talking
- coughing, wheezing, sneezing, runny nose, sore throat, fever, or signs of infection
- extreme tiredness, pain in the upper right part of the stomach, yellowing of the skin or eyes, dark yellow urine, or pale stools.
- shortness of breath
- chest pain
Delandistrogene moxeparvovec-rokl may cause liver damage and heart problems. The doctor will order blood tests to monitor your child's liver and heart before and after treatment with delandistrogene moxeparvovec-rokl injection. Talk to the doctor about the risks of your child receiving delandistrogene moxeparvovec-rokl injection.
Delandistrogene moxeparvovec-rokl may cause other side effects. Call the doctor if you have any unusual problems after receiving this medication.
If you experience a serious side effect, you or your doctor may send a report to the Food and Drug Administration's (FDA) MedWatch Adverse Event Reporting program online ( http://www.fda.gov/Safety/MedWatch ) or by phone (1-800-332-1088).
What other information should I know?
Keep all appointments with your child's doctor and the laboratory. Your doctor will order certain tests before and for at least 3 months after your treatment to check your body's response to delandistrogene moxeparvovec-rokl injection.
Delandistrogene moxeparvovec-rokl injection may only be used in children with a certain genetic make-up. Your doctor will order a blood test to help decide if this medication is right for your child.
Before having any laboratory test, tell your doctor and the laboratory personnel that your child has received delandistrogene moxeparvovec-rokl injection.
It is important for you to keep a written list of all of the prescription and nonprescription (over-the-counter) medicines you are taking, as well as any products such as vitamins, minerals, or other dietary supplements. You should bring this list with you each time you visit a doctor or if you are admitted to a hospital. It is also important information to carry with you in case of emergencies.