Status In progress
Process HST
ID number 1642

Timeline

Key events during the development of the guidance:

Date Update
13 May 2021 NICE, NHS England and NHS Improvement and PTC Therapeutics (the Company) have reached an agreement that will extend the Managed Access Agreement (MAA) for Translarna (ataluren) for treating Duchenne muscular dystrophy with a nonsense mutation in the dystrophin gene. This agreement extends the MAA, that was originally scheduled to end in July 2021, to January 2023. This will enable PTC Therapeutics to access more complete data in preparation for their resubmission to NICE and the re-evaluation of ataluren before the end of the MAA, the outcome of which will determine how ataluren will be commissioned in the long term.
09 April 2021 The Managed Access Agreement (MAA) for ataluren for treating Duchenne muscular dystrophy with a nonsense mutation in the dystrophin gene is scheduled to end in July 2021. NICE, NHS England and NHS Improvement and PTC Therapeutics (the Company) are exploring options to extend the MAA to account for the new timeline for the NICE re-evaluation, which was paused as part of NICE’s COVID-19 workplan prioritisation framework. Discussions are continuing and an update will be issued as soon as the outcome is known.
06 April 2020 In progress. Topic is in progress
06 April 2020 Topic update: this evaluation has not been defined as therapeutically critical (please follow the link titled rapid guidelines and evidence summaries within the yellow banner at the top of the page for information on recent changes to the way we work). The appraisal will therefore be paused. At this stage, we are unable to provide alternative timelines as to when the appraisal may be re-started but we will provide updates as and when this information becomes clear.

For further information on how we develop guidance, please see our page about NICE highly specialised technologies guidance