Description
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.
This product is available in the following dosage forms:
- Suspension
Before Using
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.
Pediatric
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.
Geriatric
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.
Breastfeeding
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.
Drug Interactions
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
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
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
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.
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