NICE Turns Down Translarna for Nonsense Mutation DMD

The UK’s National Institute for Health and Care Excellence (NICE) said it does not recommend PTC Therapeutics’ gene therapy Translarna (ataluren) for routine funding in the National Health Service (NHS) to treat patients with Duchenne muscular dystrophy (DMD) caused by a nonsense mutation.

The drug has been available for six years under a managed access agreement which will end in January 2023. Under the agreement NHS patients can receive the treatment while more data are being collected on its effectiveness.

NICE said the available data suggest that Translarna is likely to delay the time at which patients lose the ability to walk.  However, the evidence for improvements in later stages of the disease and improved survival is limited.

The UK list price for Translarna is approximately $245,000 per patient per year. However, if NICE did recommend the gene therapy for NHS use, it would be supplied at a discount. The agency is still in discussions with the company.