Access to treatments

EMA recommends approval of first ever treatment for all DMD patients

The European Medicines Agency (EMA) has today recommended the approval of the first ever treatment for all patients with Duchenne muscular dystrophy (DMD).

The EMA’s Committee for Medicinal Products for Human Use (CHMP) has recommended that it approves vamorolone (sold under the brand name AGAMREE) for the treatment of DMD patients aged 4 years and older (there is no upper age limit).


Vamorolone was developed as an alternative to 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 vamorolone was developed to keep or improve the positives (efficacy profile) in comparison to steroid, but with the aim of showing fewer side-effects.

Founders of Duchenne UK, Emily Reuben OBE and Alex Johnson OBE, said:

“We are delighted that the CHMP has recommended the approval of vamorolone. This is the first time in the history of Duchenne that a treatment has been approved for patients regardless of their specific mutation. Corticosteroids play a key role in treating Duchenne and we welcome as many options as possible for patients to try.”

Duchenne UK played a major role in the development of vamorolone

In 2015, Duchenne UK, Joining Jack and Duchenne Research Fund invested £750,000 towards a phase 1 clinical trial in boys with DMD, which then enabled ReveraGen to win a £6 million grant from Europe’s Horizon 2020.

Duchenne UK’s DMD Hub enabled swift recruitment to a phase 2 clinical trial in the UK, and Santhera has been engaging with us ever since to help secure access for patients in the UK.

Dario Eklund, Chief Executive of Santhera, the company developing vamorlone, said:

“We are thrilled about the CHMP’s positive opinion, which recognizes the urgent medical need for an effective and well tolerated treatment for this devastating disease. We can now execute on our plans to ensure AGAMREE is made available to patients in the EU as soon as the European Commission marketing authorization is received. The recommendation by the CHMP is a testament to the dedication and collaboration of all those involved in the development of vamorolone, including ReveraGen Biopharma, the DMD patient community, researchers, healthcare professionals, and our employees.”

Vamorolone in the UK

The EMA’s decision does not directly affect availability in the UK. However, vamorolone is under review by the Medicines and Healthcare products Regulatory Agency (MHRA) in the UK, and it will take the decision into account under the Decision Reliance Procedure.

While the National Institute for Health and Care Excellence (NICE), the regulator responsible for assessing the clinical and cost effectiveness of drugs in England, is currently undertaking a health technology assessment (HTA) for vamorolone. This will decide if it is clinically effective and cost effective for NHS patients. A decision is expected in mid 2024.

We hope that it will soon be available in the UK

We hope that it will soon be available in the UK, and we are taking part in the NICE appraisal process.

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

If you have any questions about this, you can email us at [email protected].

Published on 13 October 2023

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