Access to treatments

Vamorolone, the first treatment for all patients with DMD, approved in European Union

The European Medicines Agency (EMA) has granted marketing authorisation for vamorolone (sold under the brand name Agamree) for the treatment of Duchenne muscular dystrophy (DMD) in patients aged 4 years and older. It is approved for all people with DMD, irrespective of their underlying mutation and ambulatory (walking) status.

This makes official the EMA’s Committee for Medicinal Products for Human Use (CHMP) recommendation in October 2023 to approve vamorolone, and the Food and Drug Administration (FDA) in the US approving vamorolone in the same month for the treatment of DMD in children and adults aged 2 years and older.

Vamorolone

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.

Our role in developing vamorolone

We 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.

Our 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.

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].