NICE is unable to make a recommendation on sebelipase alfa (Kanuma) for treating lysosomal lipase deficiency that is not Wolman disease in adults. The Wolman disease population was evaluated separately in NICE highly specialised technology guidance on sebelipase alfa (HST30). The cost effectiveness of the remaining non-Wolman population has not been demonstrated at this stage.

We will review this decision if Alexion Pharmaceuticals makes a submission in the remaining non-Wolman disease population.

Guidance development process

How we develop NICE technology appraisal guidance

  • National Institute for Health and Care Excellence (NICE)