NICE publishes final guidance recommending ataluren for children with Duchenne muscular dystrophy

NICE has published final guidance recommending ataluren for treating children aged 5 and over with Duchenne muscular dystrophy (DMD) caused by a nonsense mutation.

Today's guidance follows the announcement on 7 July that NHS England and the company, PTC Therapeutics, have successfully negotiated a ‘managed access agreement’ that established financial and clinical details surrounding the use of ataluren.

This ‘managed access agreement’ was a condition for making the drug available imposed by NICE in its April draft guidance.

As part of the agreement the usual 3-month funding period has been waived by NHS England, meaning ataluren could be available within weeks.

Ataluren for treating Duchenne muscular dystrophy with a nonsense mutation in the dystrophin gene

Evidence-based recommendations on ataluren (Translarna) for treating Duchenne muscular dystrophy with a nonsense mutation in the dystrophin gene in people aged 5 years and older who can walk.