NICE recommends Translarna for treating Duchenne muscular dystrophy

The drug cost:effectiveness watchdog for England, the National Institute for Health and Care Excellence (NICE) on Friday recommended Translarna (ataluren), from US pharma company PTC Therapeutics' (Nasdaq: PTCT), for treating children aged five and over with Duchenne muscular dystrophy (DMD) caused by a nonsense mutation.

The drug has been called a ‘step change’ in the management of the disease which causes progressive muscle wasting and is usually fatal by age 30. Translana was given conditional approval in Europe in 2014, but is still awaiting clearance in the USA, where the Food and Drug Administration issued a refuse to file letter, noting that PTC’s New Drug Application was not sufficiently complete to permit substantive review (The Pharma Letter February 23). The FDA has also declined approval of BioMarin Pharmaceutical’s (Nasdaq: BMRN) NDA for Kyndrisa (drisapersen) for the treatment of Duchenne muscular dystrophy and is holding back on clearing Sarepta Therapeutics’ (Nasdaq: SRPT) eteplirsen in this indication.

Children with the disease typically become wheelchair dependent by age 12 and the committee agreed that ataluren had the potential to delay the loss of ability to walk, one of the most important factors for patients and their families.