National Institute for Health and Clinical Excellence (NICE)
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Drug company says it will negotiate terms to allow children to access new treatment for rare genetic disorder

NICE has published draft guidance that does not recommend the drug cerliponase alfa (also called Brineura and made by Biomarin) for children with neuronal ceroid lipofuscinosis type 2 (CLN2) – a very rare inherited condition affecting between 1 and 6 babies each year in the UK.

This preliminary decision, which is now the subject of public consultation until 5 March, comes as the company confirmed it is to negotiate with NHS England terms that could allow the drug to be made available while uncertainties in the evidence of its long term benefits are addressed.

Children with CLN2, which is also known as Batten Disease, live to between 8 years and early adolescence; the average life expectancy is 10 years. It is estimated that in the UK there are around 30 to 50 children living with the condition.

CLN2 is a progressive condition caused by the deficiency of the enzyme tripeptidyl peptidase 1. This results in the abnormal storage of proteins and lipids in neurons and other cells, preventing them from functioning normally.

Symptoms in children with CLN2 begin from around the age of 2 and can then progress rapidly with the onset of seizures, decline in speech, loss of mobility, involuntary muscle spasms, progressive dementia and visual impairment leading to blindness.

There is currently no cure or life-extending treatments for CLN2 and clinical management is limited to symptom relief and supportive and palliative care.

Costing over £500,000 for each year’s treatment, cerliponase alfa is an enzyme replacement therapy administered directly into the brain via a surgically implanted permanent access device.

Dr Peter Jackson, chair of the independent Highly Specialised Technology committee, said: “The committee agreed that, although cerliponase alfa is not a cure for CLN2 disease, it is an innovative treatment that is effective in the short-term in slowing the rate at which it progresses. Given the significant burden this disease places on parents and carers of children with the condition, and the subsequent negative impact this can have on the quality of their lives, anything that can help to improve the treatment of these children is to be welcomed.

“However, in the absence of long term evidence about its effectiveness in stabilising the disease and preventing death, and having taken all the health and non-health-related benefits of cerliponase alfa into account, the committee considered that the drug was not a good use of NHS resources.

“The committee welcomed the company’s intention to engage with NHS England to develop a managed access agreement to address clinical and financial uncertainties, but it concluded that it could not recommend cerliponase alfa for use in the NHS in England based on the current submission.”

Cerliponasease alfa is being considered as part of NICE’s Highly Specialised Technologies programme that looks at treatments for very rare diseases that are commissioned nationally by NHS England. 


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