NICE Guidance: Olipudase alfa for Niemann-Pick disease types AB and B

NICE Highly Specialised Technology Evaluations

Published April 2nd, 2025

Detected March 12th, 2026

Summary

NICE has published highly specialised technologies guidance (HST32) on the use of olipudase alfa (Xenpozyme) for treating Niemann-Pick disease types AB and B. The guidance provides evidence-based recommendations for healthcare professionals and commissioners in the UK.

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What changed

NICE has issued final guidance (HST32) recommending olipudase alfa (Xenpozyme) for treating acid sphingomyelinase deficiency (Niemann-Pick disease) in patients with type AB or type B. This guidance provides evidence-based recommendations for healthcare professionals, outlining the use of this highly specialised technology.

Healthcare professionals are expected to consider this guidance when making treatment decisions, in consultation with patients. Commissioners and providers are responsible for ensuring the necessary funding is available. All adverse events related to the treatment should be reported to the MHRA via the Yellow Card Scheme. The guidance will be reviewed if new evidence emerges.

What to do next

Review NICE guidance HST32 regarding olipudase alfa for Niemann-Pick disease types AB and B.
Consider olipudase alfa for eligible patients in consultation with them and/or their carers.
Ensure reporting of any adverse events to the MHRA via the Yellow Card Scheme.

Source document (simplified)

Olipudase alfa for treating acid sphingomyelinase deficiency (Niemann–Pick disease) type AB and type B

Highly specialised technologies guidance
HST32
Published:

02 April 2025

Download guidance (PDF)

Overview

Evidence-based recommendations on olipudase alfa (Xenpozyme) for treating acid sphingomyelinase deficiency (Niemann–Pick disease) in people with type AB or type B.

Last reviewed: 12 March 2025

Next review: This guidance will be reviewed if there is new evidence that is likely to change the recommendations.

Guidance development process

How we develop NICE highly specialised technologies guidance

Your responsibility

The recommendations in this guidance represent the view of NICE, arrived at after careful consideration of the evidence available. When exercising their judgement, health professionals are expected to take this guidance fully into account, alongside the individual needs, preferences and values of their patients. The application of the recommendations in this guidance is at the discretion of health professionals and their individual patients and do not override the responsibility of healthcare professionals to make decisions appropriate to the circumstances of the individual patient, in consultation with the patient and/or their carer or guardian.

All problems (adverse events) related to a medicine or medical device used for treatment or in a procedure should be reported to the Medicines and Healthcare products Regulatory Agency using the Yellow Card Scheme.

Commissioners and/or providers have a responsibility to provide the funding required to enable the guidance to be applied when individual health professionals and their patients wish to use it, in accordance with the NHS Constitution. They should do so in light of their duties to have due regard to the need to eliminate unlawful discrimination, to advance equality of opportunity and to reduce health inequalities.

Commissioners and providers have a responsibility to promote an environmentally sustainable health and care system and should assess and reduce the environmental impact of implementing NICE recommendations wherever possible.