Drug Action
Ataluren restores the synthesis of dystrophin by allowing ribosomes to read through premature stop codons that cause incomplete dystrophin synthesis in nonsense mutation Duchenne muscular dystrophy.
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Drug reference
Ataluren restores the synthesis of dystrophin by allowing ribosomes to read through premature stop codons that cause incomplete dystrophin synthesis in nonsense mutation Duchenne muscular dystrophy.
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Duchenne muscular dystrophy resulting from a nonsense mutation in the dystrophin gene, in ambulatory patients (initiated by a specialist) ▶BY MOUTH ▶Adult: (consult product literature)
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NICE decisions ▶Ataluren for treating Duchenne muscular dystrophy with a nonsense mutation in the dystrophin gene (July 2016) NICE HST3 Ataluren, within its marketing authorisation, is recommended for treating Duchenne muscular dystrophy resulting from a nonsense mutation in the dystrophin gene in people aged 5 years and older who can walk, only when: . the manufacturer provides ataluren with the discount agreed in the patient access scheme, and . the conditions under which ataluren is made available are set out in the managed access agreement between the manufacturer and NHS England, which should include the conditions set out in sections 5.12–5.15 and 5.23 of the guidance. Patients whose treatment was started within the NHS before this guidance was published should have the option to continue treatment, without change to their funding arrangements, until they and their NHS clinician consider it appropriate to stop. www.nice.org.uk/guidance/HST3 Scottish Medicines Consortium (SMC) decisions The Scottish Medicines Consortium has advised (April 2016) that ataluren (Translarna ®) is not recommended for use within NHS Scotland for the treatment of Duchenne muscular dystrophy resulting from a nonsense mutation in the dystrophin gene, in ambulatory patients aged 5 years and older as the economic case was not demonstrated. All Wales Medicines Strategy Group (AWMSG) decisions AWMSG No. 940 The All Wales Medicines Strategy Group has advised (August 2016) that the NICE HST advice for ataluren (Translarna ®) for the treatment of Duchenne muscular dystrophy resulting from a nonsense mutation in the dystrophin gene, in ambulatory patients aged 5 years and older should be implemented within NHS Wales.
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→Appendix 1: ataluren
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▶Common or very common Appetite decreased. constipation . cough. enuresis. fever. flatulence. gastrointestinal discomfort. haemorrhage. headache. hypertension. hypertriglyceridaemia. nausea. pain. skin reactions. vomiting. weight decreased ▶Frequency not known Ear infection. malaise
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Manufacturer advises avoid—toxicity in animal studies.
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Manufacturer advises discontinue breastfeeding—present in milk in animal studies.
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Manufacturer advises close monitoring—safety and efficacy not established.
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Manufacturer advises monitor renal function at least every 6–12 months, and cholesterol and triglyceride concentrations at least annually.
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Manufacturer advises the 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).
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There can be variation in the licensing of different medicines containing the same drug. Granules ▶Translarna (PTC Therapeutics Ltd) A Ataluren 125 mg Translarna 125mg granules for oral suspension sachets | 30 sachet P £2,532.00 Ataluren 250 mg Translarna 250mg granules for oral suspension sachets | 30 sachet P £5,064.00 Ataluren 1 gram Translarna 1,000mg granules for oral suspension sachets | 30 sachet P £20,256.00
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Manufacturer advises patients should maintain adequate hydration during treatment. Missed doses Manufacturer advises if a morning or midday dose is more than 3 hours late, or an evening dose is more than 6 hours late, the missed dose should not be taken and the next dose should be taken at the normal time.
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(consult product literature)
First-line fragment. Read the complete Indications and Dose section above.
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