Posted by Michael Wonder on 22 Feb 2019
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22 February 2019 - NICE has published draft guidance which confirms its earlier decision not to recommend cerliponase alfa (also called Brineura and made by BioMarin) for children with Batten disease – a very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 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.
In its earlier draft guidance, published last year, the independent committee had agreed that, although cerliponase alfa is not a cure for Batten disease (which is also called neuronal ceroid lipofuscinosis type 2), it is an important development for treating the condition and that it had shown substantial short-term benefits in slowing the rate at which it progresses.
