NICE requires further information on Translarna (ataluren) for treatment of nonsense mutation in Duchenne Muscular Dystrophy- PTC Therapeutics

The National Institute for Health & Care Excellence (NICE) in draft guidelines has sought further information from PTC Therapeutics as to the extent of benefits of Translarna (ataluren), a treatment that addresses the nonsense mutation in the dystrophin gene in children aged 5 years and older with Duchenne Muscular Dystrophy (DMD).

NICE was awaiting the results of the ACT DMD trial, however this trial (just reported) has failed to demonstrate achievement of its main goal of significantly improving the distance achieved by patients in the standard 6 minute walk test, although it showed other advantages, reported by PTC as an ability to change the course of the disease. The proposed cost of Translarna is £220,256 per patient per year.