Access to treatments

Vamorolone given PIM designation for treatment in Duchenne muscular dystrophy

October 2019

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.

Published on 20 October 2019

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About Duchenne UK

Duchenne Muscular Dystrophy (DMD) is a devastating muscle-wasting disease. It is the most common and severe form of Muscular Dystrophy. Diagnosed in childhood, it mainly affects boys. There is currently no cure. Started by families affected by the disease, Duchenne UK has one clear aim – to end Duchenne.

Duchenne UK are funding research that’s focused on getting treatments to those affected now – as well as pushing for an effective treatment in the future.

Duchenne UK connects leading researchers with industry, the NHS and patients to challenge every stage of drug development, from research to clinical trials to drug approval. They connect families with each other to create a network of mutual support and to pool resources, knowledge and experience.

How to donate

COVID-19 has caused a funding crisis in medical research. But we cannot, and will not, let it slow us down in our mission to find new and effective treatments for DMD. Because this generation of boys cannot wait. Donate now, and together we will end Duchenne

Duchenne UK is entirely reliant on donations. 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.