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.

In 2015, NICE said that BioMarin's Vimizim could be used to treat patients with the ultra-rare disease Morquio A syndrome, while real-world data was collected on its efficacy. Now – almost seven ...

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 ...