Usual Adult Dose for Muscular Dystrophy

30 mg/kg IV once a week as an IV infusion over 35 to 60 minutes

Comments:

• Use a 0.2 micron in-line filter during infusion.
• This drug is approved under accelerated approval based on an increase in dystrophin in skeletal muscle observed in some patients; a clinical benefit has not been established.
• Continued approval may be contingent upon clinical benefit in confirmatory trials.

Use: For the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 51 skipping

Usual Pediatric Dose for Muscular Dystrophy

30 mg/kg IV once a week as an IV infusion over 35 to 60 minutes

Comments:

• Use a 0.2 micron in-line filter during infusion.
• This drug is approved under accelerated approval based on an increase in dystrophin in skeletal muscle observed in some patients; a clinical benefit has not been established.
• Continued approval may be contingent upon clinical benefit in confirmatory trials.

Use: For the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 51 skipping

Renal Dose Adjustments

No adjustment recommended

Liver Dose Adjustments

Data not available

Precautions

CONTRAINDICATIONS:

• None

Consult WARNINGS section for additional precautions.

Dialysis

Data not available

Other Comments

Administration advice:

• Determine drug dosage based on patient's weight.
• Administer once weekly as an IV infusion over 35 to 60 minutes.
• A topical anesthetic cream may be applied to the infusion site prior to administration.
• Flush IV line with 0.9% Sodium Chloride Injection before and after infusion; an in-line 0.2-micron filter should be used.

Missed dose: If a dose is missed, it may be administered as soon as possible.

Preparation techniques:

• Must be diluted prior to administration.
• Allow vials to warm to room temperature.
• Mix contents of vials gently by inverting 2 or 3 times; do not shake.
• Solution should be clear and colorless, it may be slightly opalescent.
• Dilute in 0.9% Sodium Chloride for injection to make a total volume of 100 to 150 mL; infusion should be completed within 4 hours.

Storage requirements:

• Prior to use: Store at 2C to 8C (36F to 46F); Protect from light, store in original carton until ready for use.
• Diluted solution may be stored for up to 24 hours at 2C to 8C (36F to 46F); do not freeze.

IV compatibility:

• Other medications should not be mixed or infused concomitantly with this drug via the same IV line.

General:

• If a hypersensitivity reaction occurs, consider slowing the infusion or interrupting therapy.

Patient advice:

• Symptoms of hypersensitivity, including bronchospasm, chest pain, cough, tachycardia, and urticaria may occur.
• If signs and symptoms of hypersensitivity occur, seek immediate medical care.

Frequently asked questions

• Is Exondys 51 a type of gene therapy?
• Is there a specific age range for which Exondys 51 will work?
• What types of DMD can Exondys 51 be used for?
• How is Exondys 51 administered?
• What are the new drugs for Duchenne muscular dystrophy?