Pronunciation:

del-an-DIS-troe-jeen mox-e-PAR-voe-vek - rokl

Brand Names:

• Elevidys

Dosage Forms:

• Suspension

Classifications:

Therapeutic—

Musculoskeletal Agent

Uses of This Medicine:

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.

Before Using This Medicine:

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.

Children—

Appropriate studies performed to date have not demonstrated pediatric-specific problems that would limit the usefulness of delandistrogene moxeparvovec-rokl injection in children 4 years of age and older. Safety and efficacy have been established.

Older adults—

Appropriate studies have not been performed on the relationship of age to the effects of delandistrogene moxeparvovec-rokl injection in geriatric patients. Safety and efficacy have not been established.

Breast-feeding—

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.

Other medicines—

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 of This Medicine:

A nurse or other trained health professional will give you or your child this medicine in a medical facility. This medicine is given through an IV catheter that is placed into one of your veins. It must be given slowly, so the IV 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 While Using This Medicine:

Your doctor will check your or 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.

This medicine may cause infusion-related reactions. Check with your doctor right away if you have back pain, chest tightness, chills, fever, flushing, headache, nausea and vomiting, trouble breathing, or weakness.

This medicine may cause serious allergic reactions, including anaphylaxis, which can be life-threatening and require immediate medical attention. Check with your doctor right away if you have chest tightness, cough, difficulty swallowing, fast heartbeat, hives, itching, skin rash, puffiness or swelling of the eyelids or around the eyes, face, lips, or tongue, trouble breathing, or unusual tiredness or weakness.

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 of This Medicine:

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

Black, tarry stools

bleeding gums

blood in the urine or stools

pinpoint red spots on the skin

unusual bleeding or bruising

Incidence not known

Back pain

chest pain, discomfort, or tightness

cough

dark urine

dizziness

fast heartbeat

fever and chills

flushing

headache

hives, itching, skin rash

loss of appetite

muscle pain

nausea or vomiting

puffiness or swelling of the eyelids or around the eyes, face, lips, or tongue

redness or swelling in the ear

sore throat

stomach pain, severe

stuffy or runny nose

trouble breathing

unusual tiredness or weakness

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.