NICE statement on pause to re-evaluation of Translarna

As members of the Managed Access Agreement (MAA) Oversight Committee for ataluren (Translarna), Action Duchenne and Muscular Dystrophy UK have been asked to communicate the following statement to families from the National Institute for Health and Care Excellence (NICE).

As you are aware, the current MAA for ataluren (Translarna) is scheduled to end in July 2021. NICE are not in a position to be able to provide an update at present concerning the pause to the re-evaluation of ataluren in response to the Covid-19 pandemic, but we would like to reassure clinicians, patients and their families that work on this matter is in progress and we will issue communications as soon as possible in 2021 concerning the proposed next steps.

If you are a family accessing Translarna through the MAA and have any questions, please contact the Muscular Dystrophy UK helpline or email Gary Fegan at Action Duchenne.