Action Duchenne is pleased to share the announcement from Santhera Pharmaceuticals and ReveraGen BioPharma, Inc., of new results after completion of the VISION-DMD study at week 48.
VISION-DMD was a pivotal double-blind Phase 2b study designed to demonstrate efficacy and safety of vamorolone compared to placebo and prednisone (active control) in the treatment of DMD.
The FDA, USA considered both the proposed clinical efficacy and safety data of the 24-week outcomes sufficient for an NDA filing. Efficacy assessments currently reported at the completion of week 48 showed maintenance of efficacy across all parameters.
For subjects who continued on the same dose of vamorolone throughout the study, the safety profile was consistent between periods with no increase in frequency or severity of adverse events being observed over time. Subjects switching from 24-week treatment with prednisone to vamorolone 6 mg/kg/day showed no loss of efficacy through to the end of the study. In addition, vamorolone treatment at both doses (2 and 6 mg/kg/day) reversed the growth impairment observed during prednisone treatment and were associated with fewer adverse events, including those associated with corticosteroid use.
Interesting further information
Visit Duchenne explained to understand the terminology used when we talk about Duchenne research.
Read about Action Duchenne’s involvement in the Pharmacodynamics BioMarker Panel for the Phase IIa trial.
Watch the fascinating steroids session from the recent Action Duchenne International Conference ON DEMAND. Here’s how;
- Register here (FREE for all UK and International Duchenne families)
- Login using your email and create a password
- Enter the conference app
- Tap ‘Treating Duchenne and the Standards of Care’
- Move your cursor to 13 minutes 50 seconds.
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