NICE says it can approve BioMarin's ultra-rare drug treatment Vimizim, but has proposed real-world data collection and a price cap to overcome doubts about the drug's value, reports Andrew McConaghie.

Vimizim was launched in the UK with a price of £395,000 per patient per year, making it one of the most expensive drugs used by the NHS, so the initial agreement with NICE – 18 months after the ...

Though BioMarin’s portfolio comprises eight marketed products, its top line is likely to have been driven by key drugs Voxzogo, Naglazyme and Vimizim. Since its commercial launch in 2021 ...