In draft guidance published on Friday, it emerged NICE does not recommend PTC Therapeutics’ Translarna (ataluren) for the treatment of Duchenne muscular dystrophy (DMD) caused by a nonsense mutation.
The decision came following a re-evaluation of Translarna in order to decide whether to make it available for routine funding on the NHS. The re-evaluation followed a six-year period during which the DMD drug had been available under a managed access agreement (MAA), which ends in January next year, and under which 60 children have had access to Translarna.