NICE recommends Translarna (ataluren) for children aged 5 and over with Duchenne muscular dystrophy- PTC Therapeutics

The UK NICE has recommended Translarna (ataluren), from PTC Therapeutics, for treating children aged 5 and over with Duchenne muscular dystrophy (DMD) caused by a nonsense mutation. This is based on the following data: in a clinical trial, none of the children in the most sensitive group taking the drug lost the ability to walk over the 48 weeks of the trial compared with 8% on the placebo (0 out of 47 compared with 4 of 52).

In addition, the research predicted ataluren may delay loss of walking for up to 7 years. Patient experts said they had seen meaningful stabilisation or improvements in their child’s mobility such as being able to get into and out of bed independently and go to school. Finally, patient experts said once a child loses the ability to walk, greater deterioration follows meaning they need help with toileting and eating. If the time to loss of walking could be delayed, their children would have the opportunity to have a normal adolescence.

Comment: In February 2016, the FDA issued a Refuse to File letter, stating that the application was not sufficiently complete to permit a substantive review. PTC is reviewing its content to determine the appropriate next steps.