Shots:

• The P-III FoCus trial evaluates the efficacy and safety of ALXN1840 vs SoC in 214 patients aged ≥12yrs. in a ratio (3:1) with Wilson disease
• The trial met its 1EPs i.e.- improvement in daily mean copper mobilization from tissues defined as daily mean AUEC for directly measured dNCC @48wks. The therapy was generally well-tolerated with no neurological worsening- following initiation of treatment
• Alexion is working with authorities globally and plans to submit the data for review in the coming mos. The therapy has received ODD in the US and EU for the same indication

| Ref: Astrazeneca | Image: Business Standard