Shots:

• 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

  Ref: Santhera | Image: Business Wire