News

Benefits of treatment for rare genetic disorder too uncertain

Rachel Cresswell 12 February 2018

NICE has published draft Highly Specialised Technology guidance which does not recommend cerliponase alfa (Brineura, Biomarin) for children with neuronal ceroid lipofuscinosis type 2 (CLN2) – a very rare inherited condition affecting between one and six babies each year in the UK.

 

This preliminary decision, 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.

 

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

 

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