UK – Brineura hit with NICE rejection

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…