Cerliponase Alfa: Batten Disease
The question
To ask the Secretary of State for Health and Social Care, what assessment his Department has made of the potential impact of the proposals in the National Institute for Health and Care Excellence's final draft guidance, issued on 14 August 2025, to not recommend using cerliponase alfa, also called Brineura, for the treatment of children newly diagnosed with neuronal ceroid lipofuscinosis type 2, also called CLN2.
Answered by Dr Zubir Ahmed
The National Institute of Health and Care Excellence (NICE) makes its decisions independently of the Government and in line with its carefully developed methods and processes. It would not therefore be appropriate for the Department to comment on or intervene in NICE’s decision making. However, these are very difficult decisions to make, and NICE recognises that they have real and important implications for patients. It only publishes final guidance on the use of a medicine after a very careful consideration of the evidence and a consultation with stakeholders.
NICE has not yet concluded its appraisal. It has received two appeals against its draft recommendation which will be heard by NICE’s independent appeal panel on 25 November 2025. Progress of the appraisal is available at the following link:
https://www.nice.org.uk/guidance/indevelopment/gid-hst10061
Brineura is currently available to National Health Service patients for a limited period, under a managed access agreement that was agreed in 2019. This will ensure continued access for patients who have already started treatment. It will also ensure continued access for patients who begin treatment either before the agreement ends in December or when NICE publishes its final recommendations, depending on which is sooner.
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