Access to treatments

DMD treatment vamorolone approved in the US

It has been announced that the Food and Drug Administration (FDA) in the US has approved vamorolone (Agamree) for the treatment of Duchenne muscular dystrophy (DMD) in children and adults aged 2 years and older.

It is now expected to be available in the US in the first quarter of 2024.

First treatment for all DMD patients

It follows this month’s recommendation that the European Medicines Agency (EMA) approve it for the treatment of DMD patients aged 4 years and older. The EMA is expected to approve it by the end of 2023.

Vamorolone (Agamree) would then become the first drug fully approved in both the US and Europe for the treatment of all patients with DMD.

Vamorolone was developed as an alternative to corticosteroids (glucocorticoids), a routine DMD treatment to reduce muscle inflammation and maintain muscle strength, but with the aim of having fewer side-effects.

Our key role in supporting the development of vamorolone

Duchenne UK, Joining Jack and Duchenne Research Fund invested £750,000 towards a phase 1 clinical trial in boys with DMD in 2015. That enabled ReveraGen (the company developing it) to win a £6 million grant from Europe’s Horizon 2020.

Our DMD Hub supported quick recruitment to a phase 2 clinical trial in the UK, and Santhera (the company now developing it in the UK) has been engaging with us ever since to help secure access for patients in the UK.

Vamorolone in the UK

While the FDA’s approval decision does not directly affect the UK, it will be taken into account by the Medicines and Healthcare products Regulatory Agency (MHRA) in the UK, which is currently reviewing vamorolone.

The National Institute for Health and Care Excellence (NICE) is also currently undertaking a health technology assessment for vamorolone to decide if it can be available on the NHS in England. We are taking part in the process and a decision is expected in mid 2024.

We have created a process map to help you understand the approval processes for new treatments in the UK here.

Published on 27 October 2023

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