- The P-IIb VISION-DMD study involves assessing the efficacy & safety of vamorolone (2&6 mg/kg/day) vs PBO and prednisone (0.75 mg/kg/day) in 121 ambulant boys aged 4 to <7 years with DMD for 48 wks.
- The study met its 1EP i.e improvement in TTSTAND velocity, following the 1st period @24 wks. The 2 EPs include 6MWT & TTRW also showed significance over PBO, the therapy showed a favorable safety and tolerability profile over prednisone
- The company plans to file an NDA with the US FDA in Q1’22 and plans to commercialize the therapy in the US & the EU following the approval. The company has requested the PR based on the FTD granted by the FDA
Click here to read full press release/ article | Ref: Santhera | Image: Business Wire