Why is this medication 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.

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.

Other uses for this medicine

Ask your doctor or pharmacist for more information if you think this medicine could be recommended for something else.

What special precautions should I follow?

As for special precautions to follow when using delandistrogene moxeparvovec-rokl injection:

• If your kid has an allergy to any of the ingredients in delandistrogene moxeparvovec-rokl injection, any other medications, or both, let the doctor and pharmacist know. Get an ingredient list from your pharmacist.
• Inform the physician and pharmacist about any other vitamins, nutritional supplements, herbal items, prescription and over-the-counter drugs, and vitamins your child is taking or intends to take. Your doctor may need to carefully monitor you for any negative effects or adjust the dosages of your medications.
• Inform the physician if your child is infected, has heart issues, low platelet counts, or liver issues, such as cirrhosis (liver scarring) or hepatitis B or C (a virus that infects the liver and can cause severe liver damage).
• Inform the physician or dentist that your kid has received an injection of delandistrogene moxeparvovec-rokl if your child is undergoing surgery, including dental surgery.
• To find out if your child needs any immunizations, consult the doctor. It’s crucial that your child has received all vaccinations recommended for their age before starting delandistrogene moxeparvovec-rokl injectable therapy. Additionally, let the doctor know if your child has had any vaccines recently (within the last four weeks). Without first seeing their physician, your kid should not receive any vaccinations prior to or during their treatment with delandistrogene moxeparvovec-rokl injection.
• Delandistrogene moxeparvovec-rokl injection includes a virus that has the potential to spread and infect other people, therefore you should be aware of this. If your hands come into contact with your child’s bodily fluids or waste (urine, feces, saliva), you should practice good hygiene and wash your hands. For one month following the infusion, make sure to place all contaminated materials—which can include your child’s bodily fluids or waste—into a sealed plastic bag and dispose of them in the trash.

Always consult your healthcare provider if you have any questions or concerns about using Elestat or any other medication. They can provide personalized advice based on your individual medical history and needs.

What special dietary instructions should I follow?

Special dietary instructions are generally not associated with Delandistrogene moxeparvovec-rokl. However, if you have any concerns or specific dietary restrictions, it’s always best to discuss them with your healthcare provider.

What should I do if I forget a dose?

If you forget a dose of Delandistrogene moxeparvovec-rokl, take it as soon as you remember. However, if it is almost time for your next scheduled dose, skip the missed dose and continue with your regular dosing schedule. Do not double the dose to catch up. If you are unsure about what to do, consult your doctor or pharmacist for advice.

What side effects can this medication cause?

Like any medication, delandistrogene moxeparvovec-rokl can cause side effects, although not everyone experiences them. Common side effects associated with delandistrogene moxeparvovec-rokl may include:

• Nausea
• Vomiting

These side effects are usually mild and temporary. However, if any of these side effects persist or worsen, or if you experience any other unexpected symptoms while using Elestat, it’s essential to contact your healthcare provider for further guidance.

In rare cases, some individuals may experience more severe side effects or allergic reactions to delandistrogene moxeparvovec-rokl . Seek immediate medical attention if you experience any of the following:

• 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

These are not all the possible side effects of delandistrogene moxeparvovec-rokl. If you notice any other symptoms that you believe may be related to this medication, consult your doctor or pharmacist for advice. Additionally, if you have any concerns about the side effects of delandistrogene moxeparvovec-rokl, it’s important to discuss them with your healthcare provider before starting treatment.

What should I know about storage and disposal of this medication?

Storage and disposal of Delandistrogene moxeparvovec-rokl Injection:

• Storage:
  • Store Delandistrogene moxeparvovec-rokl Injection at the temperature recommended by the manufacturer. This information is typically provided on the medication label or packaging.
  • Ensure that the medication is stored in a secure location, away from children and pets.
  • Do not expose the medication to extreme temperatures or direct sunlight unless otherwise instructed.
• Disposal:
  • Follow local regulations for the proper disposal of unused or expired medications. Some pharmacies or healthcare facilities may have programs for safe disposal.
  • Do not dispose of Delandistrogene moxeparvovec-rokl Injection by flushing it down the toilet or pouring it down the drain unless instructed to do so by your healthcare provider or pharmacist.

In case of emergency/overdose

• Contact emergency medical services immediately if you suspect an overdose or if someone has injected more than the prescribed dose of Delandistrogene moxeparvovec-rokl.
• Be prepared to provide information such as the individual’s age, weight, and the amount of medication ingested.
• If possible, have the medication packaging or label available for reference.

What other information should I know?

• Side Effects: Be aware of potential side effects of Delandistrogene moxeparvovec-rokl Injection and monitor for any unusual symptoms. Common side effects may include pain or irritation at the injection site, allergic reactions, or systemic effects.
• Precautions: Inform your healthcare provider of any allergies, medical conditions, or medications you are currently taking before starting Delandistrogene moxeparvovec-rokl Injection.
• Administration: Understand the correct administration technique for Delandistrogene moxeparvovec-rokl Injection, including the proper dosage and injection site.
• Follow-Up: Follow up with your healthcare provider as directed for monitoring and evaluation of the medication’s effectiveness and any potential adverse effects.

Remember, the information provided here is general and may not apply specifically to Delandistrogene moxeparvovec-rokl Injection. Always consult your healthcare provider or pharmacist for specific instructions and guidance regarding any medication.