Usual Adult Dose for Muscular Dystrophy

Approximately 0.9 mg/kg orally once a day

Comments:

• If tablets are used, rounding up to the nearest possible dose is recommended.
• If the oral suspension is used, rounding up to the nearest tenth of a mL is recommended.
• If this drug has been administered for more than a few days, the dosage should be decreased gradually.

Usual Pediatric Dose for Muscular Dystrophy

2 years or older: Approximately 0.9 mg/kg orally once a day

Comments:

• If tablets are used, rounding up to the nearest possible dose is recommended.
• If the oral suspension is used, rounding up to the nearest tenth of a mL is recommended.
• If this drug has been administered for more than a few days, the dosage should be decreased gradually.

Renal Dose Adjustments

Mild, moderate, or severe renal dysfunction: No adjustment recommended

Liver Dose Adjustments

Mild or moderate liver dysfunction: No adjustment recommended
Severe liver dysfunction: Data not available

Comments:

• No dosing recommendation can be provided for patients with severe liver dysfunction as there is no clinical experience in these patients.

Dose Adjustments

Use with moderate or strong CYP450 3A4 inhibitors: One-third of the recommended dosage should be used.

Use with moderate or strong CYP450 3A4 inducers: Not recommended

Precautions

CONTRAINDICATIONS:

• Known hypersensitivity to the active component or any of the ingredients

Dialysis

Data not available

Other Comments

Administration advice:

• Before starting this drug, administer all immunizations according to immunization guidelines; administer live-attenuated or live vaccines at least 4 to 6 weeks before starting this drug.
• Administer with or without food.
• Do not administer with grapefruit juice.
• Tablets: Administer whole or crushed; if crushed, administer immediately after mixing with applesauce.
• Oral suspension:
• Shake well before measuring out each dose; only use the oral dispenser provided with the product.
• After withdrawing the appropriate dose into the oral dispenser, slowly add the oral suspension into 3 to 4 ounces of juice (except grapefruit juice) or milk, mix well, and then administer immediately.
• Wash the oral dispenser after each use.
• Discard 1 month after first opening the bottle.

• DMD is mostly a disease of children and young adults; there is no experience with this drug in older adults.
• In clinical trials, patients receiving 1.2 mg/kg/day demonstrated small additional benefit compared to placebo at week 12 (and no additional benefit at week 52), but had a greater incidence of adverse reactions; the use of this dose is not recommended.
• The oral suspension contains benzyl alcohol and is not approved for use in patients younger than 2 years; when prescribing the oral suspension, the combined daily metabolic load of benzyl alcohol from all sources should be considered.
• The minimum amount of benzyl alcohol that may cause serious adverse reactions is not known; at the recommended dose, patients would receive about 0.4 mg/kg/day of benzyl alcohol.

• Cardiovascular: Blood pressure
• Endocrine: For Cushing's syndrome and adrenal insufficiency (after withdrawal of this drug)
• Infections/Infestations: For the development of infection (during therapy)
• Metabolic: Blood glucose (regularly); serum potassium levels (during therapy); for signs/symptoms of volume overload (during therapy); for hyperglycemia (after withdrawal of this drug)
• Musculoskeletal: Bone mineral density (during long-term therapy)
• Ocular: Intraocular pressure (if therapy continues beyond 6 weeks)

• If prescribed the oral suspension, read the US FDA-approved patient labeling (Instructions for Use).
• Do not stop this drug abruptly or without first checking with health care providers; gradual dose reduction may be needed to reduce the risk of adrenal insufficiency.
• Inform health care provider of recent/ongoing infections or of recent vaccination; seek medical advice immediately if fever or other signs of infection develop.
• Avoid exposure to chickenpox or measles; if exposed, notify health care provider immediately.
• Seek medical attention if psychiatric symptoms (especially depressed mood or suicidal ideation) develop.
• Seek medical attention at first sign of rash.

Frequently asked questions

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