Santhera has announced that they have acquired the rights to vamorolone - a potential drug that has been developed and is currently being tested in trials by Reveragen. The trials will continue as planned and importantly, vamorolone will only be available if the trials are successful and the drug is approved by the EMA (the European medicines regulator).

Vamorolone was developed as a potential alternative to steroids - with the hope that it would have reduced side effects - currently in phase 3 trials in people living with Duchenne. Results from previous clinical studies demonstrated that there were no serious safety concerns with the drug, although some minor side effects were noted. Despite the trial not aiming to determine how effective vamorolone was, a reduction in immune system activity and a reduction in the amount of creatine kinase in the blood (a measure of muscle damage) was seen, which is encouraging.

Santhera Pharmaceuticals is the Swiss pharmaceutical company behind Raxone - a drug that is approved for the treatment of a rare eye condition called Leber's hereditary optic neuropathy - and which is currently available to people living with Duchenne, under an early access to medicines scheme in the UK.

It's important to note the vamorolone trials will continue as planned, with 3 sites in the UK. These trials will compare the new potential treatment to standard steroids and a webinar offering more details will take place on Wednesday November 28 at 6pm.

Find out more

Contact Neil Bennett, Director of Research, to find out more on 020 7250 8240

More about potential therapies for Duchenne