Deal agreed for continued access to life-changing treatment for rare inherited disease

Our draft guidance does not, at present, recommend the treatment’s use for future patients with this rare, life limiting disease due to its high price and the limited evidence of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.

With our support, 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 we publish our final recommendations or by the end of 2025, whichever is sooner.

This means that people will be able to stay on the treatment even if we are unable to recommend it for new patients beyond that point.

Alongside ourselves, NHSE and the company continue to work towards a solution to secure access to all future patients but at the moment the treatment is not considered cost effective.   

Helen Knight, director of medicines evaluation at NICE, 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.” 

The treatment has been available to NHS patients while further evidence has been collected on its use and benefits. That temporary ‘managed access period’ has been extended several times and will now end in December 2025 (or when NICE publishes its final recommendations if earlier). The additional evidence collected is being evaluated to determine whether cerliponase alfa can be used routinely on the NHS.

After reviewing new NHS data, our committee found that while cerliponase alfa clearly slows CLN2 in the short term, there’s still not enough evidence about its long-term benefits.

The committee also took into account the rarity and severity of CLN2, and the effect of cerliponase alfa on length and quality of life. It recognised the significant benefits it provides by applying a cost-effectiveness threshold 1.5 times higher than that normally applied to treatments for ultra-rare conditions and more than 5 to 8 times higher than that normally applied to treatments being evaluated outside of our HST programme.

But, because of the lack of long-term evidence of effectiveness, the price of the medicine proposed by the company is still far higher than we can consider an effective use of NHS resources.

Helen Knight continued: “We know this is not entirely the news people in the Batten disease community were hoping for. However, this is not the end of the story. We will continue to work with all parties towards a solution.”

With a list price of more than £500,000 per patient for each year’s treatment, cerliponase alfa is an enzyme replacement therapy administered directly into the brain via a surgically implanted permanent access device. 

It is estimated that in the UK there are around 30 to 50 children living with CLN2 with around 3 to 6 children newly diagnosed each year.