Delandistrogene moxeparvovec-rokl (Intravenous)

Delandistrogene moxeparvovec-rokl injection is used to treat Duchenne muscular dystrophy (DMD) in patients who have a confirmed specific genetic mutation. DMD is a rare inherited muscle disease caused by the lack of a protein called dystrophin, which helps maintain the structure of muscle cells.

This medicine should only be given by a doctor or under the direct supervision of a doctor.

Your doctor will closely monitor your child's progress while receiving this medicine to see if it is working well and to determine if you or your child should keep using it. Blood and urine tests may be required to check for unwanted effects.

Contact your doctor immediately if your child experiences pain or tenderness in the upper stomach, pale stools, dark urine, loss of appetite, nausea, unusual tiredness or weakness, or yellowing of the eyes or skin. These could be signs of a serious liver issue.

This medicine may lead to muscle problems (for example, myositis). Get in touch with your doctor right away if your child has trouble swallowing, muscle pain or weakness, difficulty breathing, or unusual tiredness or weakness.

Reach out to your doctor immediately if your child has chest pain or discomfort, fever and chills, a fast heartbeat, or trouble breathing. These may indicate myocarditis (swelling of the heart).

Your child’s vaccines should be completed at least 4 weeks before starting steroid medicine. Speak with your child’s doctor if you have any questions about this.

In deciding to use a medicine, you and your doctor must consider the risks of taking the medicine against the benefits it may provide. Here are some important points to think about for this medicine:

Allergies

Inform your doctor if you have ever had any unusual or allergic reactions to this medicine or any other medicines. Also, let your healthcare professional know if you have any other allergies, such as to foods, dyes, preservatives, or animals. For non-prescription products, carefully read the label or package ingredients.

Pediatric

There have been no appropriate studies on how delandistrogene moxeparvovec-rokl injection affects children younger than 3 years old or children 6 years old and older. Its safety and effectiveness have not been established for these age groups.

Geriatric

There is no information available about how delandistrogene moxeparvovec-rokl injection affects older adults.

Breast Feeding

There are no adequate studies in women to determine the risk to infants when using this medication while breastfeeding. Consider the potential benefits and risks before taking this medication while breastfeeding.

Interactions with Medicines

Some medicines should not be used together at all, while in other cases, two different medicines may be used together even if there could be an interaction. In these situations, your doctor may need to adjust the dose, or other precautions may be necessary. Inform your healthcare professional if you are taking any other prescription or nonprescription (over-the-counter [OTC]) medicines.

Interactions with Food/Tobacco/Alcohol

Certain medicines should not be taken around the time of eating food or certain types of food due to possible interactions. Drinking alcohol or using tobacco with certain medicines may also cause interactions. Discuss with your healthcare professional how to use your medicine with food, alcohol, or tobacco.

Other Medical Problems

Having other medical conditions may impact the use of this medicine. Be sure to tell your doctor if you have any other medical issues, especially:

• Heart disease or
• Liver disease or
• Muscle problems—Use with caution. It may worsen these conditions.

• Infection—Should be treated first before starting this medicine.

• Patients with deleted exon 8 and/or 9 in the DMD gene or
• Patients with elevated anti-AAVrh74 antibody—Use is not recommended for patients with these conditions.

Along with its necessary effects, a medicine may cause some unwanted effects. Not all of these side effects may occur, but if they do, 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

• Chest pain or discomfort
• Cough
• Dark urine
• Fast heartbeat
• Fever and chills
• Loss of appetite
• Muscle pain
• Nausea or vomiting
• Redness or swelling in the ear
• Sore throat
• Severe stomach pain
• 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.

In the U.S.

• Elevidys

Available Dosage Forms:

• Suspension

Therapeutic Class: Musculoskeletal Agent

A nurse or other trained health professional will give you or your child this medicine in a medical facility. This medicine is given through a needle placed in one of your veins. It must be given slowly, so the needle 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.