Santhera Pharmaceuticals has announced the completion of the first 6-month period of VISION-DMD, a clinical trial investigating vamorolone.
If there are positive top-line results in the data, to be released sometime in the second quarter of this year, it could lead to a submission to the Food and Drug Administration (FDA) in the first quarter of 2022. We will be monitoring this closely and encouraging Santhera to also apply in Canada, pending the outcome of the trial.
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 [3-6]. 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 DMD. There is substantial unmet medical need in this patient group as high-dose corticosteroids have significant systemic side effects that diminish patient quality of life. In the pivotal Phase 2b VISION-DMD trial , the last patient has completed the last visit of the 24-week, placebo- and active-controlled treatment period and topline 6-month data are expected in Q2-2021, paving the way for a US NDA submission in Q1-2022. 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.
Learn more about the $1M grant Defeat Duchenne Canada made towards a clinical trial evaluating vamorolone in children 2-4 years of age prior to the onset of Duchenne, and adolescents 7-18 years of age already living with Duchenne.