fbpx
Access to treatments

Interim decision: ataluren (Translarna) not recommended for NHS

Today, NICE has released draft guidance which does not recommend the use of ataluren on the NHS for the treatment of Duchenne muscular dystrophy (DMD) with a nonsense mutation in the dystrophin gene (about 15% of the DMD population). This recommendation will not affect people already taking ataluren – who may continue taking ataluren until they and their clinician consider it appropriate to stop.

Ataluren, produced by PTC Therapeutics under the brand name Translarna, is being reviewed by NICE’s Highly Specialised Technologies Committee. Ataluren is currently available on a trial basis through a managed access agreement (MAA), in place since 2016. Following a public meeting on 8 September, NICE evaluated ataluren, and today they have published their draft recommendations. You can find their evaluation consultation document (ECD) on the NICE website.

This is not the final decision. NICE is accepting comments from patient organisations and the public on the draft ECD, with a deadline for responses of 21 October. Visit the NICE website to submit comments on the draft guidance. In addition, there will be a second public meeting on 15 December. A final decision is still expected in January 2023.

NICE’s decision applies only to England, with Northern Ireland and Wales making their own decisions – though often they do the same as in England. In Scotland, ataluren will continue to be available through the Scottish Medicine Consortium’s ultra-orphan pathway.

We will be putting our concerns to NICE, and will share our response with the community.

Published on 30 September 2022

Share this article
Categories

Policy and campaigning
Policy and campaigning

NICE agrees managed access for ataluren (Translarna)

NICE has issued final guidance recommending Translarna (ataluren) for ambulatory patients aged 5 and over

Read more Read more
Access to treatments

NICE release statement on continued access to ataluren (Translarna)

NICE and NHS England have released a statement about continued access for DMD patients currently being treated with ataluren

Read more Read more
View all news