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

Is this guidance up to date?

Next review: More evidence on cerliponase alfa is being collected. After this, NICE will decide whether or not to recommend it for use on the NHS and update the guidance. It will be available through the managed access agreement until then.

Commercial arrangement

There is a managed access agreement, which includes a commercial access agreement for cerliponase alfa. Contact Biomarin at biomarin-europe@bmrn.com for details.

In March 2023, the data collection agreement was updated with changes to the information that is collected for people having cerliponase alfa through the managed access agreement. The requirement for electroencephalogram (EEG) assessments and the timing requirement for electrocardiogram (ECG) assessments were removed and adaptations for vision loss when doing neurodevelopmental assessments were clarified. For further details, please refer to table 1 (pages 4 to 6) of the data collection agreement.

Guidance development process

How we develop NICE highly specialised technology 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.

  • National Institute for Health and Care Excellence (NICE)