Cerliponase alfa for Neuronal Ceroid Lipofuscinosis Type 2 Guidance

NICE Highly Specialised Technology Evaluations

Published February 18th, 2026

Detected March 12th, 2026

Summary

NICE has published updated highly specialised technologies guidance (HST34) on cerliponase alfa (Brineura) for treating neuronal ceroid lipofuscinosis type 2 in children. This guidance replaces previous guidance and confirms that patients already receiving treatment can continue.

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What changed

NICE has issued updated guidance (HST34) concerning the use of cerliponase alfa (Brineura) for the treatment of neuronal ceroid lipofuscinosis type 2 in children. This document supersedes previous guidance (HST12) which allowed for managed access. The update confirms that individuals currently undergoing treatment with cerliponase alfa can continue their therapy until they and their healthcare professionals decide to discontinue it.

Healthcare professionals are expected to consider this guidance alongside patient needs and preferences. Commissioners and providers are responsible for ensuring funding is available for treatments recommended by this guidance. Adverse events related to the treatment should be reported to the MHRA via the Yellow Card Scheme. The guidance itself does not impose new compliance deadlines or penalties, but emphasizes the responsibility of healthcare providers and commissioners.

What to do next

Review updated NICE guidance HST34 regarding cerliponase alfa.
Continue existing cerliponase alfa treatments as per patient and clinician decision.
Report any adverse events related to cerliponase alfa to the MHRA Yellow Card Scheme.

Source document (simplified)

Cerliponase alfa for treating neuronal ceroid lipofuscinosis type 2

Highly specialised technologies guidance
HST34
Published:

18 February 2026

Download guidance (PDF)

Overview

Evidence-based recommendations on cerliponase alfa (Brineura) for treating neuronal ceroid lipofuscinosis type 2 in children.

Last reviewed: 18 February 2026

This guidance updates and replaces NICE's highly specialised technologies guidance 12 on cerliponase alfa for treating neuronal ceroid lipofuscinosis type 2, which was available with managed access. People already taking it will be able to continue until they and their healthcare professional decide when best to stop.

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.