NICE does not recommend cerliponase alfa to treat ceroid lipofuscinosis type 2 disease.- Biomarin.
The National Institute for Health and Care Excellence (NICE) has published draft guidance that does not recommend the drug cerliponase alfa (also called Brineura and made by Biomarin) for children with neuronal ceroid lipofuscinosis type 2 (CLN2) �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 . 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. 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, NICE considered that the drug was not a good use of NHS resources.
Learning Zones
The Learning Zones are an educational resource for healthcare professionals that provide medical information on the epidemiology, pathophysiology and burden of disease, as well as diagnostic techniques and treatment regimens.
2 mins