Posted by Michael Wonder on 14 Aug 2025
Final decision on cerliponase alfa for Batten disease treatment confirmed
14 August 2025 - In final draft guidance published today we've confirmed that we’re unable to recommend cerliponase alfa (Brineura; BioMarin) for routine NHS use in treating neuronal ceroid lipofuscinosis type 2, a rare and life-limiting form of Batten disease.
This means cerliponase alfa will not be made available to children newly diagnosed with ceroid lipofuscinosis type 2. However, during the course of its evaluation, we supported commercial discussions between NHS England and BioMarin. These resulted in a separate agreement that will ensure continued access for patients who have already started treatment or who begin treatment before the most recent managed access agreement extension ends in December 2025 or when we publish our final recommendations, whichever is sooner.
