Translarna Fast Track - access to treatments

Translarna (also known as ataluren) is a treatment for people with Duchenne muscular dystrophy with a nonsense genetic mutation, manufactured by pharmaceutical company PTC Therapeutics. It is currently available to eligible children aged 2 and over with Duchenne muscular dystrophy through a Managed Access Agreement (MAA) in England (which also covers Wales and Northern Ireland) and through the ultra-orphan pathway in Scotland.

In May 2021, NICE announced that the MAA had been extended from June 2021 to January 2023, by which point they will have made a full assessment of whether it should be provided on the NHS in the longer-term. In Scotland, access through the ultra-orphan pathway is available until early 2024.

MDUK is a member of the Managed Access Oversight Committee (MAOC) for Translarna and will be working with the Duchenne community to fully engage in both the NICE and SMC processes.