The National Institute for Health and Care Excellence (NICE) Highly Specialised Technology committee is supporting a positive recommendation for BioMarin's cerliponase alfa, brand name Brineura▼ for children with neuronal ceroid lipofuscinosis type 2 (CLN2) (also known as Batten's disease) – a very rare inherited condition affecting between one and six babies each year in the UK.
The decision results from a managed access agreement made with the manufacturer. Managed access schemes are agreements between the manufacturer and the NHS, usually specifying a set of conditions under which reimbursement for the medicine will be made.
Cerliponase alfa is an enzyme replacement therapy administered directly into the brain via a surgically implanted permanent access device.
The independent committee noted that although cerliponase alfa is not a cure for CLN2 disease, it is an important development for treating the condition, and that it has shown substantial short-term benefits in slowing the rate at which it progresses.
You can follow developments with Brineura by using our Medicines Tracker service which provides users with updates about the medicines they are interested in.
About Brineura (source EMA)
Brineura is a medicine for treating neuronal ceroid lipofuscinosis type 2 (CLN2 disease), an inherited condition in children that leads to progressive brain damage.
Because the number of patients with CLN2 is low, the disease is considered ‘rare’, and Brineura was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 13 March 2013.
Patients with CLN2 lack an enzyme required for normal brain development called tripeptidyl-peptidase-1 (TPP1). The active substance in Brineura, cerliponase alfa, is a copy of TPP1 and is used as a replacement for the missing enzyme.
The medicine is infused directly into the brain in order to bypass the blood-brain barrier, a protective barrier that separates the blood stream from the brain, and prevents substances such as medicines, from entering brain tissue.
Brineura was first made available in the EU in 2017. It is manufactured by BioMarin International.
European Medicines Agency website page for Brineura
Links available in External Resources
© NICE 
Cerliponase alfa for treating neuronal ceroid lipofuscinosis type 2 [ID943]. In development [GID-HST10008]. Expected publication date: 27 November 2019
Available from: See Link below. All rights reserved. Subject to Notice of rights
NICE guidance is prepared for the National Health Service in England. All NICE guidance is subject to regular review and may be updated or withdrawn. NICE accepts no responsibility for the use of its content in this product/publication.
The information provided by NICE was accurate at the time this article was issued.
NICE information on Brineura
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About Batten's disease (Neuronal ceroid lipofuscinosis)
About Neuronal ceroid lipofuscinosis