NICE again says no to BioMarin's Batten disease drug

22 February 2019

The UK’s medicines cost-effectiveness watchdog has published draft guidance which confirms its earlier decision not to recommend Brineura (cerliponase alfa), made by US biotech firm BioMarin Pharmaceutical (Nasdaq: BMRN), for children with Batten disease – a very rare inherited condition affecting between one and six babies each year in the UK.

The National Institute for Health and Care Excellence (NICE) 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.

Although no new price details were mentioned, when the NICE rejected Brineura last year it said that the drug cost nearly £523,000 ($726,000) for a year’s treatment, based on list price of £20,107 for a 300mg pack.

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