Research funded by Duchenne UK produces the first ever drug for treatment of everyone with Duchenne muscular dystrophy (DMD)
It’s not enough to discover and trial a new drug. It must be approved by regulators too.
Now this final step has taken place, it’s expected that the first ever treatment for all DMD patients will become available in 2024.
This breakthrough is the direct result of research and trials funded by Duchenne UK over the last decade.
Vamorolone, sold under the brand name Agamree, has been approved in the United
States for patients from the age of 2 years old, and approved in Europe for patients aged 4 and up.
In the UK, we are engaging the National Institute for Health and Care Excellence (NICE), which will decide if the new drug can be made available on the NHS in England, to support a decision in mid 2024.
Currently, DMD patients are given steroids to reduce the progression of the disease. But this comes at a cost; weight gain, delayed puberty and osteoporosis. Duchenne UK wanted better for DMD children.
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 BioPharma to win a £6 million grant from Europe’s Horizon 2020 fund.
Two years later, we helped to set up the Phase 1 trial through the DMD Hub, offering ReveraGen assistance and support.
The data showed the drug to be safe, and the company was able to go to Phase 2 trials, again supported by Duchenne UK, which were also successful.
Santhera Pharmaceuticals hopes to bring the drug to market in Europe in the first half of 2024.
A landmark year of achievement and impact