ReveraGen BioPharma, the developer of vamorolone, received a 3.3 million dollar grant from the National Institutes of Health (NIH) to support the commercialization of this safer alternative for corticosteroids treatment. This will advance vamorolone towards FDA submission and commercialization in the USA. This is excellent news for the Duchenne community and is still pending their final results anticipated in Q2 of 2021. We continue to work with ReveraGen to find solutions that will accelerate this program in Canada.
Defeat Duchenne Canada has funded $1 million towards the VBP15-006 clinical trial program in Canada, evaluating vamorolone in very young children aged 2-4 and older boys aged 7-18. We are very excited about this progress and will continue to update you on this program. If you have any questions regarding this information or the vamorolone clinical trial program, please contact:
Director of Research and Advocacy
Vamorolone is a first-in-class drug candidate that binds to the same receptor as corticosteroids but modifies its downstream activity and as such is a dissociative partial agonist. This mechanism has the potential to ‘dissociate’ efficacy from typical steroid safety concerns and therefore vamorolone could emerge as a promising alternative to existing corticosteroids, the current standard of care in children and adolescent patients with Duchenne.
There is a substantial unmet medical need in this patient group as high-dose corticosteroids have significant systemic side effects that diminish the patient’s quality of life. The fully enrolled, pivotal Phase 2b VISION-DMD trial (VBP15-004) is currently being conducted at study sites across North America, Europe, Israel and Australia and topline 6-month data are expected in Q2-2021, paving the way for a US NDA submission in Q4-2021.
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 and Promising Innovative Medicine (PIM) status from the UK MHRA. Vamorolone was discovered by US-based ReveraGen BioPharma, Inc. and is being developed in collaboration with Santhera, which owns worldwide rights to the drug candidate in all indications. The vamorolone development program has received funding from several international non-profit foundations and patient organizations, the US National Institutes of Health, the US Department of Defense and the European Commission’s Horizon 2020 program: https://vision-dmd.info.
About ReveraGen BioPharma
ReveraGen was founded in 2008 to develop first-in-class dissociative steroidal drugs for Duchenne muscular dystrophy and other chronic inflammatory disorders. The development of ReveraGen’s lead compound, vamorolone, has been supported through partnerships with foundations worldwide, including Defeat Duchenne Canada, Muscular Dystrophy Association USA, Parent Project Muscular Dystrophy, Foundation to Eradicate Duchenne, Save Our Sons, JoiningJack, Action Duchenne, CureDuchenne, Ryan’s Quest, Alex’s Wish, DuchenneUK, Pietro’s Fight, Michael’s Cause, and Duchenne Research Fund. ReveraGen has also received generous support from the US Department of Defense CDMRP, National Institutes of Health (NCATS, NINDS, NIAMS), and European Commission (Horizons 2020).