The UK National Institute for Health and Care Excellence (NICE) has published a draft guidance saying that Biomarin’s therapy for Batten disease, Brineura (cerliponase alfa), cannot be recommended for use in the National Health Service (NHS).
Batten disease, which is also known as neuronal ceroid lipofuscinosis type 2, is a rare inherited condition.
Brineura is an enzyme replacement therapy that is directly administered into the brain through a surgically implanted permanent access device.
NICE issued a similar guidance in February last year, noting that Brineura has substantial short-term benefits in slowing the disease, but lacked evidence on its long-term effectiveness in stabilising the condition and preventing death.
The cost-effectiveness watchdog said that even after year-long negotiations between Biomarin and NHS England, the company failed to propose the therapy’s price at a level that would address the problems highlighted during its assessment.
In addition to the absence of long-term evidence, NICE also reviewed all health and non-health-related benefits of Brineura and concluded that therapy’s price is not acceptable in terms of what the organisation usually considers as normal for highly specialised technologies.
NICE Centre for Health Technology Evaluation director Meindert Boysen said: “We and NHS England have been very clear with the company about what would be needed in order for us to be able to recommend cerliponase alfa.
“However, despite being given ample opportunity to come up with a workable solution, regrettably the company has not been able to do so.”
Caused due to deficiency of the tripeptidyl peptidase 1 enzyme, Batten disease affects one to six babies each year in the UK. Children with this condition are known to have an average life expectancy of ten years.
The disease could lead to seizures, decline in speech, loss of mobility, involuntary muscle spasms, progressive dementia, and visual impairment and ultimately blindness.