To ask His Majesty's Government what assessment they have made of the impact of ineligibility to receive cerliponase alfa for treatment of neuronal ceroid lipofuscinosis type 2 (CLN2) on children born after January 2026; and what steps they are taking to ensure equitable access to treatment for all future patients who are diagnosed with CLN2.
Answered on
1 July 2025
In the absence of treatment with cerliponase alfa, clinical management of patients with neuronal ceroid lipofuscinosis type 2 (CLN2) focuses on symptom control, monitoring and preventing complications, and palliative care. The aim is to maintain function for as long as possible and to improve quality of life. Following the next National Institute for Health and Care Excellence Appraisal Committee meeting in July, the National Institute for Health and Care Excellence 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.