NICE: Biomarin’s Batten Disease Treatment is Not Cost-Effective

The UK’s National Institute for health and Care Excellence (NICE) determined that Biomarin’s Brineura is not cost-effective for Batten disease patients.

Batten disease, which affects one in six babies in the UK, causes abnormal protein storage in patients, leading to progressive dementia and visual impairment. Average life expectancy is only 10 years.

Biomarin’s enzyme replacement therapy, while effective at slowing the condition’s progress in the short term, did not demonstrate long-term stabilizing effects, according to NICE.