Breaking: Update on Translarna NHS treatment for Duchenne muscular dystrophy

Published Date
13/05/2021
Author
Alice Cachia
Category
Research

NHS England and NHS Improvement, in partnership with NICE, has today announced that the Managed Access Agreement (MAA) for the drug Translarna has been extended for use on NHS England until January 2023.

Translarna can help treat a version of Duchenne muscular dystrophy that is caused by nonsense mutations.

Any new patients in England who are eligible for the treatment will be able to start accessing it during the extended period of the MAA.

How did we get to this point?

MDUK has been working in partnership with Action Duchenne and families affected by Duchenne muscular dystrophy to campaign for access to Translarna.

Since the MAA began in 2016 we have represented the Duchenne community on the NICE-led Managed Access Oversight Committee.

What happens now?

We have started gathering questions from the Duchenne community and are working with NICE to share comprehensive information about the extension and what it means. We will be publishing a first set of Q&As later today (13 May).

NICE’s website statement on the announcement can be read here: https://www.nice.org.uk/guidance/hst3

What do we think?

Representing patients on the Managed Access Oversight Committee, Robert Burley, Director of Campaigns, Care and Support at MDUK, and Shelley Simmonds, an advocate for the Board of Trustees at Action Duchenne said:

“Today’s announcement is welcome news and will be a relief to the families who were waiting anxiously for confirmation that the Managed Access Agreement would be extended from July 2021 to January 2023. It is vital that all parties now work together to ensure that the right decision can be made by the end of the extension period, and that throughout the next 18 months there is clear communication with the Duchenne community. We will continue to support families while working as part of the Managed Access Oversight Committee.”

What’s happening elsewhere?

Last month the Scottish Medicines Consortium accepted Translarna for use on NHSScotland for three years through the ‘ultra-orphan pathway’. More information can be read in our news story here.

Those wanting to reach our support team can get in touch by ringing our helpline 0800 652 6352 between 10am-2pm on Monday to Friday. Alternatively, you can email us on info@musculardystrophyuk.org

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