Delandistrogene moxeparvovec-rokl Injection

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 adults and children 4years of age and older. It is in a class of medications called gene therapy. It works by replacing a defective gene in muscles which helps improve muscle function.

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 injection may cause serious reactions during the infusion or up to about 3 hours after the infusion. A doctor or nurse will monitor you during the infusion and for 3 hours after your infusion is finished to see if you are having a serious reaction to the medication. Tell your doctor or nurse immediately if you experience any of the following symptoms: fast or racing heart beat; shortness of breath or fast breathing; swelling of the face, throat, tongue, lips, or eyes; flushing; hives; itching; rash; fever; chills; nausea; vomiting; or difficulty swallowing or breathing.

Delandistrogene moxeparvovec-rokl may cause serious immune response reactions after you receive the infusion. The doctor will prescribe a corticosteroid (a type of medication used to prevent swelling and inflammation) for you 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 you misss a corticosteroid dose or vomit after taking a dose of corticosteroids.

Ask your pharmacist or doctor for a copy of the manufacturer's information for the patient.

This medication may be prescribed for other uses; ask your doctor or pharmacist for more information.

Before receiving delandistrogene moxeparvovec-rokl injection,

• tell the doctor and pharmacist if you are 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 you are 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 you have an infection or if you have or have 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.
• tell your doctor if you are pregnant, plan to become pregnant, or are breast-feeding. Delandistrogene moxeparvovec-rokl is not intended for use in pregnant women.
• if you are having surgery, including dental surgery, tell the doctor or dentist that you have received delandistrogene moxeparvovec-rokl injection.
• check with the doctor to see if you need to receive any vaccinations. It is important to have all vaccines appropriate for your age before beginning treatment with delandistrogene moxeparvovec-rokl injection. Also tell the doctor if you have recently (within 4 weeks) received any vaccinations. You should not have any vaccinations before or after your 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 bodily fluids or waste (urine, feces, saliva). You should be sure to put all contaminated materials that may contain your bodily fluids or waste into a sealed plastic bag and throw them away into the garbage for 1 month after the infusion.

Unless your doctor tells you otherwise, continue your normal diet.

Delandistrogene moxeparvovec-rokl injection may cause side effects. Tell the doctor if any of these symptoms are severe or do not go away:

• nausea
• vomiting

Some side effects can be serious. If you experience any of these symptoms or those listed in the HOW section, 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 liver and heart before and after treatment with delandistrogene moxeparvovec-rokl injection. Talk to the doctor about the risks of 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 (https://www.fda.gov/Safety/MedWatch) or by phone (1-800-332-1088).

Keep all appointments with your 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 people with a certain genetic make-up. Your doctor will order a blood test to help decide if this medication is right for you.

Before having any laboratory test, tell your doctor and the laboratory personnel that youhave 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.

• Elevidys^®