NICE says drug to treat Batten disease cannot be recommended for NHS use because company is unable to address concerns about long-term effectiveness

22 February 2019 – NICE

NICE has published draft guidance confirming its earlier decision not to recommend cerliponase alfa (also called Brineura and made by Biomarin) for children with Batten disease – a very rare inherited condition affecting between 1 and 6 babies each year in the UK.

The decision was made 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.

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