UK MHRA assigns PIM status to ReveraGen’s Vamorolone as a DMD therapy

Duchenne muscular dystrophy (DMD) approximately affects 1 in every 3,500 male births every year in the world. several biopharmaceutical and biotechnology firms across the globe are increasingly working towards the discovery and development of novel therapies for this disease.

One of the contributors to this effort is Santhera Pharmaceuticals, a renowned Swiss pharmaceutical firm focused at the commercialization and development of medicines for DMD (Duchenne muscular dystrophy), reportedly announced that ReveraGen Biopharma, a US based pharmaceutical firm, has been awarded the PIM (Promising Innovative Medicine) designation for their vamorolone drug by the UK MHRA (Medicines and Healthcare products Regulatory Agency) to treat DMD (Duchenne muscular dystrophy).

CEO of Santhera, Thomas Meier, PhD stated that Santhera congratulates ReveraGen on its success in finding a possible treatment for DMD and the company is thrilled about the PIM designation grant as it validates the capabilities of vamorolone as an advanced treatment approach which would address the high unmet medical requirement in young patients suffering from DMD.

The PIM designation granted by MHRA signifies that the agency considers vamorolone as a hopeful candidate for EAMS (Early Access to Medicines Scheme). In the United Kingdom, PIM is the primary step of EAMS, which aims to provide patients with seriously debilitating or life-threatening conditions access to those medicines which don’t have a marketing authorization yet if there is a strong unmet medical need.     

ReveraGen’s Vamorolone is a steroidal, anti-inflammatory first-in-class investigational drug that is under development as a DMD treatment. Data from clinical and non-clinical studies specified that treatment with vamorolone showed a persistent improvement of muscle function along with lesser adverse effects, generally reported for traditional corticosteroids.

Vamorolone drug has received Orphan Drug status in Europe and the U.S. Furthermore, the drug has also received designations of Fast Track and Rare Pediatric Disease by the U.S. FDA.     

The currently under progress Phase IIb VISION-DMD 48 week trial (VBP15-004; NCT03439670) is specifically designed like a pivotal study to showcase safety and efficacy of vamorolone in comparison to placebo and prednisone in 120 boys aged 4 to below 7 suffering from DMD who have not been treated with corticosteroids yet.

Source credit: http://www.santhera.com/assets/files/press-releases/2019-10-21_PIM_Vamorolone_e_final.pdf