Santhera and ReveraGen announce positive topline results with Vamorolone after completion of the VISION-DMD study. As previously reported, the FDA considered the safety and efficacy data of vamorolone at 24 weeks (period 1) sufficient for an NDA filing.
- Vamorolone given throughout the study showed sustained efficacy across multiple endpoints over 48 weeks and the good safety and tolerability profile was confirmed
- Switching from prednisone to vamorolone after week 24 maintained efficacy and improved on multiple safety parameters including restoration of growth trajectory and reduction of behavioral changes up to week 48
- Switching from placebo to vamorolone after week 24 resulted in an improvement of multiple efficacy outcome parameters with no apparent increase in the rate of treatment-emergent adverse events (TEAE) up to week 48
- Study confirmed good safety and tolerability profile of vamorolone with 98% subjects completing the period from week 24 to week 48
On Wednesday December 1st, 15:00 – 16:00 CET, you are invited to join families, researchers, patient organizations and clinicians to the online end event and discussion of this project.
Efficacy assessments at the now reported completion at week 48 (period 2) included Time to Stand (TTSTAND) velocity, 6-Minute Walk Test (6MWT), Time to Run/Walk 10 meters (TTRW) velocity, and North Star Ambulatory Assessment (NSAA). Vamorolone 6 mg/kg/day showed maintenance of efficacy across all parameters until end of study at week 48, and was statistically superior to 2 mg/kg/day for TTSTAND velocity and 6MWT but not for TTRW velocity or NSAA.
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 reversed the growth impairment seen during prednisone treatment and was associated with fewer adverse events, including those associated with corticosteroid use.