New medicine for DMD, funded by Duchenne UK and patient groups, becomes first approved treatment in the UK for all patients with DMD
The National Institute for Health and Care Excellence (NICE) has approved vamorolone for use on the NHS to treat Duchenne muscular dystrophy (DMD). Vamorolone, also known by the brand name Agamree, will be available to treat people with DMD aged four years and older. We have nurtured the development of vamorolone for ten years in a ground-breaking example of venture philanthropy.
Alternative to existing corticosteroids
Vamorolone, produced by Santhera Pharmaceuticals, was created as an alternative to existing 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 some DMD patients do not tolerate it well or at all. Vamorolone now gives their neuromuscular consultant a new treatment option for them.
Duchenne UK’s role in developing vamorolone
Fueled by a desire to provide more treatment options for DMD, we invested in and supported vamorolone’s development from the initial laboratory research through to its health technology assessments.
Duchenne UK, along with our partner charities, Joining Jack and Duchenne Research Fund, invested £750,000 towards a Phase 1 clinical trial in boys with DMD in 2015. We then advised on trial protocol and funded the clinical research site for the vamorolone Phase 2 trial and the Principal Investigator. This then enabled ReveraGen to win a £6 million grant from Europe’s Horizon in 2020 to support the development of vamorolone.
Our Project HERCULES, a multinational collaboration set up to develop tools and evidence to support health technology assessments, provided crucial evidence about the impact of DMD to build the case for patient access to it to.
Our founders, who both have teenage sons with DMD, welcome the NICE announcement. Emily Reuben OBE, Chief Executive of Duchenne UK, and Alex Johnson OBE, Chief Executive of Joining Jack, said:
“We were given very little hope and few treatment options when our sons were diagnosed with DMD. We could not and would not accept that and have raised millions of pounds for research into new treatment options for DMD, one of which was vamorolone. We are very proud that a treatment that we have supported for so long will now be available in the NHS. The approval of vamorolone is the culmination of a global effort of scientists, clinicians and patient advocacy groups investing in and supporting the development of vamorolone. The venture philanthropy model of our investment means any money returned to us will be reinvested into research for new DMD treatments.”
Vamorolone in the UK
Vamorolone was approved by the UK’s Medicines and Healthcare products Regulatory Agency (MHRA) in January this year, but was initially rejected by NICE for use in NHS England in March this year.
NICE’s new decision to approve it will apply within NHS England which must make sure it is as a treatment option available within three months. As NICE guidance is usually adopted in Wales and Northern Ireland, it is expected to be soon available there too. The Scottish Medicines Consortium (SMC) is currently appraising vamorolone for use on the NHS.
NHS Adoption
Our DMD Care UK programme will be publishing information materials for patients and families about vamorolone and steroid use to treat DMD in early 2025.
We will also be discussing this at our New Horizons conference that’s taking place in London from Friday 28 – Saturday 29 March 2025. You can find out more about it and register for a place here.