NICE statement on Translarna

Published Date
Jonathan Kingsley
Image of the five boys who went to Downing Street in 2015 campaigning for the drug Translarna

Muscular Dystrophy UK and Action Duchenne have received the following public statement from NICE on ataluren (Translarna) which we can share with the community, as follows:

“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.”

We would like to provide re-assurance to you that Muscular Dystrophy UK and Action Duchenne jointly are continually and regularly communicating with NICE on this very sensitive issue for families and will provide further updates as soon as we can.

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