Question to the Department of Health and Social Care:

To ask His Majesty's Government whether they plan to revisit the Managed Access Agreement for Brineura for treating neuronal ceroid lipofuscinosis type 2 to ensure that children born after January 2026 are not excluded from the treatment.

The National Institute for Health and Care Excellence (NICE) is currently updating its guidance on the use of cerliponase alfa (Brineura) for the treatment of neuronal ceroid lipofuscinosis type 2 (CLN2) and has not yet published final guidance. NICE published its draft guidance for consultation in May 2025 and was unfortunately unable to recommend cerliponase alfa for routine use when taking into account all the evidence of costs and benefits, including real world evidence collected during the managed access period. We are, however, pleased that NICE and NHS England have been able to reach an agreement with the manufacturer of cerliponase alfa that ensures continued access to treatment for all patients who started on treatment before December 2025.

Officials in the Department have been in regular contact with NICE and NHS England about this appraisal. Following the next NICE Appraisal Committee meeting in July, NICE and NHS England will continue to try to reach an agreement with the manufacturer based on the committee's preferred assumptions around modelling that will provide access to cerliponase alfa for all future patients.

It is estimated that in the UK, approximately three to six children are diagnosed with CLN2 each year. In the absence of treatment with cerliponase alfa, clinical management options focus on symptom control, monitoring and preventing complications, and palliative care. Management aims to maintain function as long as possible and to improve quality of life.