PPMD is pleased to share the announcement from Santhera Pharmaceuticals and ReveraGen BioPharma, Inc., of new topline 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.
As previously reported, the FDA considered the safety and efficacy data of vamorolone at 24 week (period 1) 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.
To view Santhera’s letter to the community click here.
To learn more about the study click here.