TRANSLARNA Granules for oral suspension Ref.[7623] Active ingredients: Ataluren

Treatment with Translarna should only be initiated by specialist physicians with experience in the management of Duchenne/Becker muscular dystrophy.

Posology

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).

Translarna is available in sachets of 125 mg, 250 mg or 1000 mg. The table below provides information on which sachet strength(s) to use in the preparation of the recommended dose by body weight range.

Delayed or missed dose

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.

Special populations

Elderly

The safety and efficacy of ataluren in patients aged 65 and older have not yet been established (see section 5.2).

Renal impairment

No dosage adjustment is required for patients with mild or moderate renal impairment. Treatment of patients with severe renal impairment (eGFR <30 ml/min) or end-stage renal disease is not recommended (see sections 4.4 and 5.2).

Hepatic impairment

No dosage adjustment is required for patients with mild, moderate or severe hepatic impairment (see section 5.2).

Paediatric population

Paediatric patients with body weight ≥12 kg are treated as per the dosing recommendations by body weight range (see above dosing table). 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 Translarna in children <12kg and aged 6 months to 2 years have not yet been established. No data are available.

Method of administration

Translarna should be administered orally after mixing it to a suspension in liquid or in semi-solid food. 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.

For instructions on reconstitution of the medicinal product before administration, see section 6.6.

Healthy volunteers receiving a single oral dose of 200 mg/kg of ataluren experienced transient, low-grade symptoms of headache, nausea, vomiting, and diarrhoea. No serious adverse reactions were observed in these subjects. In the event of a suspected overdose, supportive medical care should be provided including consulting with a healthcare professional and close observation of the clinical status of the patient.

4 years.

Each prepared dose is best administered immediately after preparation. The prepared dose should be discarded if not consumed within 24 hours of preparation if kept refrigerated (2–8°C), or within 3 hours at room temperature (15–30°C).

This medicinal product does not require any special storage conditions.

For storage conditions after dilution of the medicinal product, see section 6.3.

Heat-sealed laminated aluminium foil sachet: polyethylene terephthalate (child resistance), polyethylene (colouring and polyester/foil bond), aluminium foil (moisture barrier), adhesive (polyurethane class), copolymer of ethylene and methacrylic acid (sealant resin for packaging integrity).

Pack of 30 sachets.

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.

Any unused medicinal product or waste material should be disposed of in accordance with local requirements.