Posted by Michael Wonder on 20 Jul 2016
NICE publishes final guidance recommending ataluren for children with Duchenne muscular dystrophy
20 July 2016 - NICE has published final guidance recommending ataluren for treating children aged 5 and over with Duchenne muscular dystrophy 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This ‘managed access agreement’ was a condition for making the drug available imposed by NICE in its April draft guidance.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.
