Active Ingredient: Ataluren
Ataluren is indicated for the treatment of Duchenne muscular dystrophy resulting from a nonsense mutation in the dystrophin gene, in ambulatory patients aged 2 years and older.
The presence of a nonsense mutation in the dystrophin gene should be determined by genetic testing.
For this indication, competent medicine agencies globally authorize below treatments:
For:
Oral, 40 milligrams ataluren per kilogram of body weight, divided daily to meals, 3 doses in total.
Ataluren should be administered orally every day in 3 doses.
The first dose should be taken in the morning, the second at midday, and the third in the evening. Recommended dosing intervals are 6 hours between morning and midday doses, 6 hours between midday and evening doses, and 12 hours between the evening dose and the first dose on the next day.
The recommended dose is 10 mg/kg body weight in the morning, 10 mg/kg body weight at midday, and 20 mg/kg body weight in the evening (for a total daily dose of 40 mg/kg body weight).
If there is a delay in the administration of ataluren of less than 3 hours after the morning or midday doses or less than 6 hours after the evening dose, the dose should be taken with no changes to the subsequent dose schedules. If there is a delay of more than 3 hours after the morning or midday doses or more than 6 hours after the evening dose, the dose should not be taken, and patients should resume their usual dosing schedule. Patients should not take a double or extra dose if a dose is missed. It is important to administer the correct dose. Increasing the dose above the recommended dose may be associated with reduced effectiveness.
Paediatric patients with body weight ≥12 kg are treated as per the dosing recommendations by body weight range. The recommended dose is the same for all age ranges, i.e. 10 mg/kg body weight in the morning, 10 mg/kg body weight at midday, and 20 mg/kg body weight in the evening (for a total daily dose of 40 mg/kg body weight).
The safety and efficacy of ataluren in children <12kg and aged 6 months to 2 years have not yet been established. No data are available.
Ataluren should be administered orally after mixing it to a suspension in liquid or in semi-solid food. Ataluren sachets should only be opened at the time of dose preparation. The full contents of each sachet should be mixed with, at least 30 ml of liquid (water, milk, fruit juice) or 3 tablespoons of semi-solid food (yoghurt or apple sauce). The prepared dose should be mixed well before administration. The amount of the liquid or semi-solid food can be increased based on patient preference. Patients should take the entire dose.
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