3.1 Ataluren (Translarna, PTC Therapeutics) 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 (DMD). Ataluren has a conditional marketing authorisation in the UK for treating DMD resulting from a nonsense mutation in the dystrophin gene in patients aged 5 years and older who can walk. The continuation of marketing authorisation is linked to analysis by the European Medicines Agency of results provided from a phase III trial (Study 020).
3.2 The summary of product characteristics lists the most frequent adverse reactions as nausea, vomiting and headache (occurring in 1 in 10 people or more). For full details of adverse reactions and contraindications, see the summary of product characteristics.
3.3 The recommended dosage of ataluren is 40 mg/kg body weight per day. The company submission states that the list price of ataluren is £2,532 per box of 30 sachets containing ataluren 125 mg. Assuming a median weight range of 24 kg to 26 kg, the total cost per person per year of treatment with ataluren is £220,256. The company has agreed a patient access scheme with the Department of Health. This scheme provides a simple discount to the list price of ataluren with the discount applied at the point of purchase or invoice. The level of the discount is commercial in confidence. The Department of Health considered that this patient access scheme does not constitute an excessive administrative burden on the NHS. The proposed managed access agreement includes further confidential financial components that would apply while it is in force.