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Access to treatments

Vamorolone approved for use in NHS Scotland

Duchenne muscular dystrophy (DMD) treatment vamorolone has been approved for use in NHS Scotland. The Scottish Medicines Consortium (SMC) announced today that vamorolone, also known by the brand name Agamree, will be available to treat people with DMD aged four years and older.

New treatment option

It follows last month’s approval by the National Institute for Health and Care Excellence (NICE) for it to be used as a DMD treatment in NHS England.

Vamorolone, produced by Santhera Pharmaceuticals, was created as an alternative to existing corticosteroids, also called glucocorticoids, which are routinely used in the treatment of DMD as they can reduce muscle inflammation and maintain muscle strength. Steroid treatment has several negative side-effects, and some DMD patients do not tolerate it well or at all, so vamorolone now gives their neuromuscular consultant a new treatment option for them.

Duchenne UK’s role in developing vamorolone

Duchenne UK has invested in and supported vamorolone’s development from the initial laboratory research through to its health technology assessments. Along with our partner charities, Joining Jack and Duchenne Research Fund, we invested £750,000 towards a Phase 1 clinical trial in boys with DMD in 2015. We advised on trial protocol and funded the clinical research site for the vamorolone Phase 2 trial and the Principal Investigator. This then enabled ReveraGen to win a £6 million grant from Europe’s Horizon in 2020 to support the development of vamorolone from.
 
Our Project HERCULES, a multinational collaboration set up to develop tools and evidence to support health technology assessments, provided crucial evidence about the impact of DMD to build the case for patient access to it to.

Our DMD Care UK programme will be publishing information materials for patients and families about vamorolone and steroid use to treat DMD in early 2025.

We will also be discussing this at our New Horizons conference that’s taking place in London from Friday 28 – Saturday 29 March 2025. You can find out more about it and register for a place here.

Published on 13 January 2025

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