DMD study in vamorolone reports positive results
pharmafile | June 1, 2021 | News story | Manufacturing and Production |
Santhera Pharmaceuticals and ReveraGen BioPharma have announced positive results from their Phase IIb VISION-DMD study, evaluating the efficacy and safety of vamorolone compared to placebo and prednisone (active control) in the treatment of Duchenne muscular dystrophy (DMD).
The study met its primary endpoint of superiority in change of time to stand from supine positioning to standing (TTSTAND) velocity with vamorolone 6mg/kg/day, versus placebo. This corresponds to a clinically relevant improvement in TTSTAND in the vamorolone 6 mg/kg/day group from 6.0 to 4.6 seconds and a corresponding deterioration in the placebo group from 5.4 to 5.5 seconds. The study also demonstrated superiority of vamorolone versus placebo across multiple secondary endpoints.
Craig McDonald, Professor and Chair of the Department of Physical Medicine & Rehabilitation and Director of Neuromuscular Disease Clinics, UC Davis Health, said: “The strength of evidence for both efficacy and safety of vamorolone over such a wide dose range from 2 to 6 mg/kg/day allows clinicians to individually tailor treatment of Duchenne patients by starting at the higher 6 mg/kg/day dose of vamorolone with equivalent efficacy to daily prednisone and titrate the dose according to how well the treatment is tolerated whilst maintaining optimal efficacy.
“I am enthusiastic that this approach may allow patients to avoid side effects that currently lead to discontinuing steroid treatment, meaning they are able stay on for longer.”
Santhera plans to submit a New Drug Application (NDA) in the US in 2022, requesting a priority review based on the fast track designation granted by the FDA. The VISION-DMD study continues to 48 weeks and will, subject to a positive outcome, deliver data for the submission of a marketing authorisation application in Europe next year.
The trial also highlighted that vamorolone does not cause stunting of growth, a common side effect of conventional corticosteroids.
Paula Clemens, study Co-Chair and Vice Chair of VA Affairs and Professor of Neurology, University of Pittsburgh School of Medicine, said: “Stunting of growth is a major concern of families and patients treated with corticosteroids, and we are delighted to see this superiority of vamorolone proven in this double-blind study.”
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 FDA and Promising Innovative Medicine (PIM) status from the UK MHRA.
Kat Jenkins
