NICE shot down Biomarin’s Brineura (cerliponase alfa), a treatment for children with Batten disease, citing concerns about the treatment’s long-term effectiveness and pricing.
After a year of price negotiations between the company and NHS England, NICE decided against recommending the drug, noting that the national health service could not accept the drugmaker’s complicated commercial deal.
“The cost-effectiveness estimates for cerliponase alfa are far higher than NICE normally considers acceptable for highly specialised technologies,” the institute said, noting that the drug also lacks evidence of effectiveness in stabilizing the disease and preventing death.
Batten disease, a very rare inherited neurodegenerative disorder, affects between one to six babies every year in the UK.
