We are delighted to welcome the news that the Medicines and Healthcare products Regulatory Agency (MHRA) has approved vamorolone (sold under the brand name Agamree) for the treatment of Duchenne muscular dystrophy (DMD) in patients aged four years and older.

This is the first treatment approved for all people with DMD, irrespective of their underlying mutation and ambulatory (walking) status.

This is a key milestone towards access to vamorolone for patients and families in the UK. However, it is not yet available on the NHS.

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 and cost effective for NHS patients. A decision is expected in mid-2024.

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, Joining Jack, Duchenne UK 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.

Our co-founder, Alex Johnson OBE, Chief Executive of Joining Jack, and Emily Reuben OBE, Chief Executive of Duchenne UK, welcomed the news:

“We are delighted that the first drug developed to benefit everyone with Duchenne has been approved in the UK.

“When our sons were diagnosed with Duchenne, we were told that steroids, the standard medication for children with it, would keep them independently mobile for longer. But with harmful side effects.

“We didn’t think this was good enough, and invested in finding better treatments.

“Partner charities Duchenne UK, Joining Jack and the Duchenne Research Fund, funded the early-stage clinical research to test vamorolone in patients, when no-one else would. For it now to be available in the UK to treat DMD is proof that we can find better treatments for Duchenne and change things for Duchenne.”