The National Institute for Health and Care Excellence (NICE) says a drug to treat Batten disease cannot be recommended for NHS use, because the company is unable to address concerns about long-term effectiveness .

Brineura (cerliponase alfa) is a treatment for neuronal ceroid lipofuscinosis type II (CLN2) - also known as Batten disease, which costs over £500,000 per person for each year’s treatment.

The enzyme replacement therapy is administered directly into the brain via a surgically implanted permanent access device.

The decision comes at the end of year-long negotiations between the company and NHS England, during which the company was unable to price the treatment at a level that would have addressed the problems highlighted during NICE’s assessment of it.

Meindert Boysen, director of the Centre for Health Technology Evaluation at NICE, said: “We and NHS England have been very clear with the company about what would be needed in order for us to be able to recommend cerliponase alfa. However, despite being given ample opportunity to come up with a workable solution, regrettably the company has not been able to do so.

“The committee concluded that it is not possible within the NHS to manage complex components of the confidential commercial arrangement proposed by the company. Once these elements are excluded, the cost-effectiveness estimates for cerliponase alfa are far higher than NICE normally considers acceptable for highly specialised technologies. We are therefore left with no alternative but to conclude that cerliponase alfa cannot be recommended as a cost-effective use of NHS resources.”

The disease is a progressive condition caused by the deficiency of the enzyme tripeptidyl peptidase 1, which results in the abnormal storage of proteins and lipids in neurons and other cells, preventing them from functioning normally.