The National Institute for Health and Care Excellence has published draft guidance that does not currently recommend the routine NHS use of cerliponase alfa (Brineura) for newly diagnosed patients with neuronal ceroid lipofuscinosis type 2 (CLN2), a rare and life-limiting form of Batten disease.
The decision is based on the treatment’s high cost—over £500,000 per patient per year—and limited long-term effectiveness data.
In a significant move to protect those already receiving the treatment, NICE, NHS England, and manufacturer BioMarin have agreed that patients who begin treatment before the end of 2025—or before final guidance is published—will continue to receive Brineura permanently. This ensures that no child currently benefiting from the therapy will be forced to stop, even if the treatment is not approved for future use.
Brineura is an enzyme replacement therapy administered directly into the brain via a surgically implanted device. It has been shown to slow the progression of CLN2 in the short term, but NICE’s committee concluded that evidence of long-term benefit remains insufficient.
Despite recognising the severity of CLN2 and applying a cost-effectiveness threshold 1.5 times higher than usual for ultra-rare diseases, the committee found that the proposed price still exceeds what can be considered a good use of NHS resources.
What’s Next?
- The managed access period has been extended until December 2025 or until final guidance is published.
- NICE, NHS England, and BioMarin continue to work toward a long-term solution to secure access for future patients.
- The public consultation on the draft guidance is open until 6 June 2025, with a final decision expected later this year.
Director of Medicines Evaluation at NICE, Helen Knight, said:
“We’re pleased that NICE has been able to support NHSE and the company in reaching an agreement to make access to cerliponase alfa permanent for everyone who has already started treatment and those who will start treatment before the managed access agreement ends in December. NICE, together with NHS England, remains committed to working with the company to try to reach a long-term deal that will give access to cerliponase alfa to all eligible people after that time.”
With only 30 to 50 children in the UK living with CLN2 and 3 to 6 new diagnoses annually, the outcome of this consultation will have a profound impact on families facing this devastating condition.
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