We’re happy to announce that the MHRA (the medicines regulator in the UK) has given Promising Innovative Medicine (or PIM) status to Vamorolone as a treatment for Duchenne Muscular Dystrophy. Designating a drug as a Promising Innovative Medicine is the first step in the Early Access to Medicines scheme.
In the UK, the Early Access to Medicines Scheme (EAMS) is a regulatory scheme that aims to give patients with life threatening or seriously debilitating conditions access to medicines that do not yet have a marketing authorisation when there is a clear unmet medical need. Vamorolone has now taken the first step in this process – the next step will be an assessment of the potential benefits and risks of the drug by the MHRA.
The development of Vamorolone was supported by Action Duchenne, alongside many other UK and international charities and other research funders. The drug was designed to offer some of the effectiveness of steroids with reduced side effects. It has shown encouraging results in early trials and its effectiveness is now being tested in a large trial that is currently enrolling patients age 4 to 7 years at 6 sites in the United Kingdom (in Newcastle, Glasgow, Liverpool, Leeds, London and Birmingham.
Any questions?
- email Neil or call us on 02072508240
- Come along to the Action Duchenne conference
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