Final decision on cerliponase alfa for Batten disease treatment confirmed
In final draft guidance published today we've confirmed that we’re unable to recommend cerliponase alfa (also called Brineura and made by BioMarin) for routine NHS use in treating neuronal ceroid lipofuscinosis type 2 (CLN2), a rare and life-limiting form of Batten disease.
In final draft guidance published today we've confirmed that we’re unable to recommend cerliponase alfa (also called Brineura and made by BioMarin) for routine NHS use in treating neuronal ceroid lipofuscinosis type 2 (CLN2), a rare and life-limiting form of Batten disease.
This means cerliponase alfa will not be made available to children newly diagnosed with CLN2. 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.
Today’s final draft guidance comes after the company’s decision not to enter into commercial negotiations with NHS England following our appraisal committee meeting last month. As a result, the price charged by the company does not represent a cost-effective use of NHS resources.
The medicine is currently available on the NHS in England for a limited period under a managed access agreement (MAA). This enables NHS patients to access promising medicines that demonstrate plausible cost-effectiveness for a fixed period, at a discount, while further evidence is gathered on how these medicines can be provided and how well they work outside clinical trials. MAAs help to address clinical uncertainties for the NHS and are not considered routine commissioning
NHS England, supported by us, negotiated a series of extensions to the MAA, most recently in April 2025, during which time all eligible patients have continued to be able to access the medicine. Cerliponase alfa’s time in MAA – six years – has been among the longest agreements of its kind, providing as much opportunity as possible for additional data to be collected and assessed.
We are deeply disappointed that the company decided not to enter into commercial negotiations with NHS England having reached an agreement that maintains continued access for patients who were started on treatment during the managed access period. The company’s decision means that the committee was not presented with a cost-effective price that would have allowed newly diagnosed children to benefit from this treatment.
Jonathan continued: “Throughout this process the company has had multiple opportunities to reconsider the price of cerliponase alfa. While the medicine has been shown to be effective in slowing disease progression, the price the company chose is simply too high for NICE to recommend its use in the NHS – even when applying a cost-effectiveness threshold ten times higher than usual.”
Cerliponase alfa is an enzyme replacement therapy administered directly into the brain, with an annual list price exceeding £500,000 per patient.
In the UK, it is estimated that 30 to 50 children are currently living with CLN2, with around 3 to 6 new diagnoses each year.
Stakeholders, including patient groups and the company, now have the opportunity to appeal the final draft guidance. If no appeals are received NICE expects to publish its final recommendations on cerliponase alfa next month.