First ever treatment for ALL Duchenne patients is recommended for approval

Vamorolone, the first ever treatment for ALL DMD patients, has been recommended for approval by the European Medicines Agency, and it’s thanks to you, our incredible supporters.

It was developed as an alternative to corticosteroids, which are routinely used in the treatment of Duchenne Muscular Dystrophy as they can reduce muscle inflammation and maintain muscle strength.

Thanks to your support, Joining Jack played a major role in the development of Vamorolone. In 2015, alongside Duchenne UK and The Duchenne Research Fund, we invested £750,000 towards a phase 1 clinical trial in boys with DMD, which then enabled ReveraGen to win a £6million grant from Europe’s Horizon 2020.

Our DMD Hub enabled swift recruitment to a phase 2 clinical trial in the UK, and Santhera has been engaging with us ever since to help secure access for patients in the UK.

Through your support and donations Vamorolone is the second biggest investment Joining Jack and our charity partners Duchenne UK has made.

I remember talking to Prof Katie Bushby about some good early data on a compound called Vamorolone, when she asked would we consider funding the Phase 1 trial? Emily and I approached our friend Kerry Rosenfeld at the Duchenne Research Fund, and so between the three charities we funded the Phase 1. This was critical.

We bridged what’s known as the notorious ‘valley of death’ in drug development where promising compounds languish in labs because no one is prepared to fund the risky early phase 1 trials due to the high likelihood of failure.

The data from Phase 1 was positive and this enabled the company to secure a 5 million euro grant from EU Horizon funding to run Phase 2/3 trials. Emily and I have been closely involved in the development, and helped the company on the clinical trial protocol.

This is not a gene therapy and it does not address the underlying cause of Duchenne. It is good to have another option for corticosteroids if boys struggle with Deflazacort and Prednisolone.

At this stage we are unsure of what this means for Jack and Emily’s son, Eli, because it has only been tested it on younger boys. Trials are ongoing in older boys, so we will wait for that data, and the next milestone is to work with NICE to get it reimbursed for the UK.

However, this is a great day for the community and we are very proud to have played a big part in it. Thank you for all your help and support in helping us make this happen.’

You can read more about the decision here