October 2019

  • Vamorolone has been designated Promising Innovative Medicine (PIM) for treatment in Duchenne muscular dystrophy.
  • This is the initial step towards access to the drug on the Early Access to Medicines Scheme (EAMS).

Vamorolone is a drug being developed by ReveraGen Biopharma, which could provide an alternative to the current standard of care for DMD, glucocorticoid steroids. 

Duchenne UK, along with Joining Jack, and The Duchenne Research Fund, made a grant of $1million to support the Phase 1 trial of vamorolone. Muscular Dystrophy America also invested in this project.

In 2014 we campaigned for – and won - a change in the law to allow patients with life limiting conditions to access as yet un-approved drugs through the EAMS. Today – more than 46 boys with DMD in the UK are now taking a drug called Raxone because of that scheme which is helping to improve and stabilise their lung function. Thanks to the hard work of Duchenne UK, the Duchenne community and other UK Duchenne patient organisations, we are delighted to see the scheme being used for a treatment for DMD.

In the UK the EAMS is a regulatory path by the MRHA that aims to give patients with life threatening or seriously debilitating conditions access to medicines that do not yet have a marketing authorization when there is a clear unmet medical need. The initial step in this process is Promising Innovative Medicine (PIM) designation.

Co-founders of Duchenne UK Alex Johnson and Emily Crossley said,

“We are delighted that MHRA has given PIM status to Vamorolone as a treatment for Duchenne Muscular Dystrophy. A PIM designation is the first step of a process that could allow patients earlier access to a new medicine. This is part of the Early Access to Medicines Scheme (EAMS) which Duchenne UK and Joining Jack lobbied for in 2014. We are pleased to see that the scheme may be used for Vamorolone.”

UK foundations that have aided the development of vamorolone for DMD include Joining Jack, Duchenne UK (formerly Duchenne Children’s Trust), ActionDuchenne, Alex’s Wish Foundation, and Duchenne Research Fund.

Vamorolone is a first-in-class drug that targets multiple biochemical pathways in DMD patient muscle simultaneously, and in initial open label studies has shown improvements of patient muscle function. A pivotal trial that may lead to drug approval is currently enrolling patients age 4 to 7 years at 6 sites in the United Kingdom (Newcastle University, Royal Hospital for Children [Glasgow], Alder Hey Children's Hospital [Liverpool], Leeds Teaching Hospital Trust, Great Ormond Street Institute of Child Health [London] and University Hospitals Birmingham). Information on the currently recruiting vamorolone clinical trial with contact information for UK recruitment sites can be found on the DMD Hub Clinical Trial Finder: https://dmdhub.org/trials/vamorolone-phase-2b/.

About the UK Early Access to Medicines Scheme (EAMS)

The UK's industry-sponsored EAMS aims to give patients with life threatening or seriously debilitating conditions access to medicines that do not yet have a marketing authorization when there is a clear unmet medical need. The EAMS is a two-step process:

Step I is the Designation as a Promising Innovation Medicine (PIM). The PIM designation is an early indication that a medicinal product is a promising candidate for EAMS and gives reassurance that its clinical development is on track by having an early review of its data by the medicines regulator.

Step II is the Scientific Opinion by the Medicines and Healthcare products Regulatory Agency (MHRA, UK regulatory agency). The Scientific Opinion describes the benefits and risks of the medicine and supports the prescriber and patient to make a decision on using the medicine before its license is approved.




What is Duchenne Muscular Dystrophy?

Duchenne muscular dystrophy (DMD) is the most common fatal genetic disease diagnosed in childhood. Children born with DMD cannot produce the protein dystrophin which is vital for muscle strength and function. Muscle weakness starts in early childhood. Many use a wheelchair by around the age of 12. As deterioration continues it leads to paralysis and early death, often in their 20s. It almost exclusively affects boys. There is no treatment or cure. In the UK there are around 2,500 boys affected and around 300, 000 worldwide. It is classified as a rare disease.

Who are Duchenne UK?

Duchenne UK is a lean, ambitious and highly focused charity with a clear vision: to fund and accelerate treatments and a cure for Duchenne muscular dystrophy.

We are investing millions of pounds in research right now to bring treatments and a cure to help this generation. Duchenne UK is the largest funder of DMD research in the UK. We are also committed to accelerating the pace of research. 90p in every £1 raised is committed to research.

Our president is HRH The Duchess of Cornwall. Our patrons include the broadcasters Krishnan Guru-Murthy and Mary Nightingale, and the sports stars Owen Farrell, Kris Radlinski and Andy Farrell. 

We need your help, because we need to keep funding promising new research.

How to donate?

Duchenne UK is entirely reliant on donations to fund research for treatments and a cure to DMD. This can be done via:

  • Direct Debit – Duchenne Direct
  • Individual Donation – Donate
  • If you are a family or friend affected by DMD you can set up your own fund with Duchenne UK – Family and Friend Funds
  • Take part in one of our fundraising events – Events
  • Text DUCHENNE to 70085 to donate £5. This costs £5 plus a std rate msg.

For more information visit www.duchenneuk.org