Update on vamorolone for treatment of Duchenne muscular dystrophy

Santhera Pharmaceuticals has announced that the UK’s Medicines and Healthcare products Regulatory Agency (MHRA) has designated vamorolone as Promising Innovative Medicine (PIM) for the treatment of Duchenne. This is the first of several steps in the Early Access to Medicine Scheme (EAMS), which aims to give people living with debilitating conditions access to medicines that are not yet licensed.

Vamorolone is a steroid-like drug that is thought to have fewer side effects than current steroid treatments for Duchenne. A placebo-controlled phase 2b trial comparing vamorolone to prednisolone is currently recruiting in the UK.

You can read the press release from Santhera Pharmaceuticals here.


Previous news related to this story:

11 October 2019

Duchenne trial open for recruitment in UK

A phase 2b trial testing vamorolone is now recruiting at six UK sites (Newcastle, Glasgow, Liverpool, Leeds, Birmingham and London). Vamorolone is a steroid-like drug that has fewer side effects than current steroid treatments for Duchenne muscular dystrophy. More information about the trial and contacts at each trial site can be found on clinicaltrials.gov

For more information, read this press release from the VISION-DMD research group. 

28 August 2019

Results from vamorolone trial published

Results from a six-month study testing vamolorone in boys with Duchenne have been published in the academic journal, Neurology. Vamorolone is an anti-inflammatory drug that could be an alternative to steroids. The study showed that vamorolone was safe and improved muscle function in a dose-dependent manner.

Vamorolone is currently being tested in a global phase 2b trial called VISION-DMD, which has UK sites including London, Newcastle, Liverpool, Glasgow and Leeds (Birmingham is not yet open for recruitment). Eligibility criteria and contact details for UK sites can be found on clinicaltrials.gov

7 September 2016 

Duchenne steroid alternative being trialled in the UK

ReveraGen BioPharma has today announced that enrollment has begun for its phase 2a trial testing vamorolone (VBP15) in boys with Duchenne muscular dystrophy. Vamorolone is an anti-inflammatory drug that is similar to steroids but may have fewer side effects.

Trial participants must be steroid-naïve (no current or previous treatment with steroids) and aged between 4-6 years old. The UK arm of the trial will take place at the Newcastle-upon-Tyne Hospitals NHS Trust.

For more information about the trial, please visit http://vision-dmd.info/2a-trial-information/ or read the company’s press release.


15 March 2016

Million pound boost for Duchenne research

The European Commission awarded The John Walton Muscular Dystrophy Research Centre at Newcastle University £4.6 million for a new clinical trial to test the anti-inflammatory drug, vamorolone for the treatment of Duchenne muscular dystrophy.

For more information, read Newcastle University’s press release.

To find out more about the latest research into DMD, please contact our Research Line at research@musculardystrophyuk.org or call 020 7803 4813.