Shots:

• NICE has issued final draft guidance recommending elosulfase alfa for routine use in the NHS as a treatment option for the treatment of mucopolysaccharidosis type 4A
• The recommendation was based on the real-world clinical data from 69 patients treated with Vimizim (elosulfase alfa) which showed the long-term benefits with elosulfase alfa treatment indicating deceleration of MPS 4A progression
• Elosulfase alfa has been developed and marketed by BioMarin Pharmaceutical under the trade name Vimizin. Additionally, MPS 4A leads to a reduction in life expectancy in an inherited lysosomal storage disease

Ref: NICE | Image: BioMarin