NICE recommends new drug for Duchenne muscular dystrophy to be made available in England
10 December 2024
Today (Tuesday 10 December) NICE (the National Institute for Health and Care Excellence) has recommended that the drug vamorolone (also sold under the brand name Agamree) be made available for people aged four years and over with Duchenne muscular dystrophy in England. This is a big step forward in the availability of treatment options for the condition and provides an alternative to the corticosteroids that are currently prescribed.
We played a leading role in the NICE appraisal of vamorolone, working in partnership with other Duchenne charities. We nominated and supported Mandy Roe as a patient expert to share her 12-year-old son’s experience of receiving the treatment through a clinical trial. Our Director of Care, Campaigns and Support, Rob Burley, also served as a patient expert to the appraisal process and represented the views of the Duchenne community, which we gathered through extensive engagement.
This is great news for families as vamorolone provides an alternative choice to corticosteroids. NICE heard evidence about the acute side effects that can be experienced when taking these and the comparatively less severe ones associated with vamorolone.
Mandy’s son TJ was diagnosed with Duchenne aged three. As part of a clinical trial he took vamorolone between the ages of six to 11 years old. She said: “When I found out vamorolone was going to be recommended by NICE I was ecstatic. Although we were very lucky TJ was able to receive vamorolone, clinical trials take their toll on children and families like mine.
It was amazing to be able to tell TJ that we did it; everything he’d been through had helped make this drug available to thousands of others.
Mandy
“All the drives in the dark of the night, all the tears and pain and exhaustion, had been worth it to get to this point.”
We are proud to have played a key role in ensuring vamorolone is available for people with Duchenne.
Rob Burley
“Throughout the appraisal process, we heard firsthand about the dilemma that families face when it is recommended that their child starts taking corticosteroids. The associated side effects, including weight gain, bone weakness and mood changes, cause huge concern and the community shared powerful testimony about their experiences and the benefit that an alternative treatment option would bring.”
Integrated Care Boards and NHS England must comply with a NICE recommendation within three months of its date of publication. This means that, if a patient has Duchenne muscular dystrophy and the healthcare professional responsible for their care thinks that vamorolone is the right treatment, it should be available for use by the end of March 2025.
When NICE recommends the use of a drug or treatment, the NHS in Wales must usually provide funding and resources for it within two months of the publication of the final draft guidance. Northern Ireland also generally follows NICE guidance. In Scotland, we have also played a leading role in the Scottish Medicines Consortium appraisal process and they are expected to publish their guidance on vamorolone in January 2025.
If you have any questions about eligibility for the treatment, speak with your clinical team.
“Vamorolone helped my son maintain his independence”
Mandy’s 12-year-old son, TJ, who has Duchenne muscular dystrophy, received Vamorolone for six years via a clinical trial. She believes he gained huge benefits on the drug. In celebration of Vamorolone being recommended by NICE (National Institute for Health and Care Excellence) on 10 December, Mandy tells their story. She shares their personal experience of how they feel Vamorolone helped TJ, and what happened when he came off the treatment and switched to standard use corticosteroids.
For people with muscle wasting or weakeningconditions, every day is vital which is why we’re committed to ensuring urgent action is taken so that people are able to access the support and treatments that they want and need.
