Ataluren for treating Duchenne muscular dystrophy with a nonsense mutation in the dystrophin gene

Ataluren (Translarna, PTC Therapeutics) has a conditional marketing authorisation 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 dosage schedule is available in the summary of product characteristics.

The price for ataluren is £2,532 per box of thirty 125‑mg sachets, £5,064 per box of thirty 250‑mg sachets and £20,256 per box of thirty 1,000‑mg sachets (excluding VAT; BNF online accessed September 2022).

The company has a commercial arrangement. This makes ataluren available to the NHS with a discount. The size of the discount is commercial in confidence. It is the company's responsibility to let relevant NHS organisations know details of the discount.