Posted by Michael Wonder on 12 Feb 2018
Drug company says it will negotiate terms to allow children to access new treatment for rare genetic disorder
12 February 2018 - NICE has published draft guidance that does not recommend the drug cerliponase alfa (Brineura; Biomarin) for children with neuronal ceroid lipofuscinosis type 2 – a very rare inherited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 ceroid lipofuscinosis type 2, 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.
