BRIEF—BioMarin's Batten disease drug now to be made available, says NHS England

NHS England and US biotech firm BioMarin have reached agreement that will now make Brineura (cerliponase alfa) available for eligible children in England with the fatal, progressive condition, Late Infantile Batten Disease (CLN2).

Confirmation of the deal to bring the treatment onto the National Health Service (NHS) follows negotiation between NHS England and BioMarin, which has agreed a fair price that the National Institute for Health and Care Excellence (NICE) committee was able to recommend as an effective use of NHS resources.

In February 2019, the NICE published draft guidance not to recommend Brineura for children with Batten disease due to the absence of long-term data and therefore considered that the drug was not value for money within the context of a highly specialised service.

In August 2019, the NICE and BioMarin met again to discuss NHS access to Brineura in England. This decision has followed that meeting.