“We congratulate ReveraGen on this success and are excited about the PIM designation as it further validates the potential of vamorolone as an innovative treatment approach addressing the high unmet medical need in young patients with Duchenne muscular dystrophy,” said Thomas Meier, PhD, CEO of Santhera.
The PIM designation indicates that the UK MHRA considers vamorolone a promising candidate for the Early Access to Medicines Scheme (EAMS). In the UK, the EAMS, of which PIM is the first step, 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.
Vamorolone is a first-in-class steroidal anti-inflammatory investigational drug in development as treatment for DMD. Data from non-clinical and clinical studies indicated that vamorolone treatment results in a persistent improvement of muscle function with less adverse effects typically reported for traditional corticosteroids.
Vamorolone has been granted Orphan Drug status in the US and in Europe and has received Fast Track and Rare Pediatric Disease designations by the US FDA.
About Vamorolone – first-in-class dissociative steroid
Vamorolone is a first-in-class drug candidate that binds to the same receptors as corticosteroids but modifies the downstream activity of the receptors. This has the potential to ‘dissociate’ efficacy from typical steroid safety concerns and therefore could replace existing corticosteroids, the current standard of care in children and adolescent patients with DMD. There is significant unmet medical need in this patient group as high dose corticosteroids have severe systemic side effects that detract from treatment compliance and patient quality of life.
Vamorolone is being developed by US-based ReveraGen BioPharma Inc. with participation in funding and design of studies by several international non-profit foundations, the US National Institutes of Health, the US Department of Defense and the European Commission’s Horizon 2020 program. In November 2018, Santhera acquired from Idorsia the option to an exclusive sub-license to vamorolone in all indications and all countries worldwide (except Japan and South Korea).