Continued access to treatment for type of Batten Disease

The National Institute for Health and Care Excellence are consulting on draft guidance for cerliponase alfa for treating neuronal ceroid lipofuscinosis type 2 (CLN2), a type of Batten disease. The draft guidance does not, at present, recommend the treatment’s use for future patients with this rare, life limiting disease due to its high price and the limited evidence of long-term effectiveness. NHS England and BioMarin have agreed to permanently provide cerliponase alfa (also known as Brineura) to people with CLN2 who have already started treatment or will be started on treatment before the end of the newly extended access period. This is due to close when final recommendations are published or by the end of 2025, whichever is sooner.