NICE has published draft guidance stating that although Translarna (also known as ataluren) is clinically effective and an innovative treatment, there is doubt as to its cost effectiveness. NICE’s draft recommendation is that people currently receiving Translarna should continue to have access to it until they and their NHS clinician consider it appropriate to stop, but that it shouldn’t be provided for new patients after January 2023. This continuing access for existing patients is welcome, but we are very disappointed by this draft decision regarding new patients and are urging NICE and PTC Therapeutics to work together to address the concerns raised about cost effectiveness.

We recently announced that NICE has approved new treatment, avalglucosidase alfa for Pompe Disease. This is one of ten treatments across several neuromuscular conditions that MDUK has worked on with NICE and the SMC through their appraisal processes over the past three years.