The National Institute for Health and Care Excellence (NICE) have recommended that cerliponase alfa, brand name Brineura▼, as being suitable for patients with neuronal ceroid lipofuscinosis type 2.
This means that the medicine is available to patients by NHS England and Wales.
Neuronal ceroid lipofuscinosis type 2 (CLN2 disease), is an inherited condition in children that leads to progressive brain damage.
CLN2 disease is also known as Batten's disease.
NICE state: 'Cerliponase alfa is recommended as an option for treating neuronal ceroid lipofuscinosis type 2 (CLN2), also known as tripeptidyl peptidase 1 (TPP1) deficiency, only if the conditions in the managed access agreement are followed.
This recommendation is not intended to affect treatment with cerliponase alfa that was started in the NHS before this guidance was published. People having treatment outside this recommendation may continue without change to the funding arrangements in place for them before this guidance was published, until they and their NHS clinician consider it appropriate to stop. For children or young people, this decision should be made jointly by the clinician and the child or young person, or the child's or young person's parents or carers.
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.
You can follow developments on 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 2016. It is manufactured by BioMarin.
European Medicines Agency website page for Brineura
Links available in External Resources
© NICE 
Cerliponase alfa for treating neuronal ceroid lipofuscinosis type 2. Highly specialised technologies guidance [HST12]. Published 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: Cerliponase alfa for treating neuronal ceroid lipofuscinosis type 2
New medicines and vaccines that are under additional monitoring have an inverted black triangle symbol (▼) displayed in their package leaflet and summary of product characteristics, together with a short sentence explaining what the triangle means – it does not mean the medicine is unsafe. You should report all suspected adverse drug reactions (ADRs) for these products. ADRs can be reported by your doctor, pharmacist or online via the Yellow Card
Reporting of suspected adverse reactions
Reporting suspected adverse reactions (side effects) after authorisation of the medicinal product is important. It allows continued monitoring of the benefit/risk balance of the medicinal product. Healthcare professionals or patients are asked to report any suspected adverse reactions via the Yellow Card Scheme at yellowcard.mhra.gov.uk or search for MHRA Yellow Card in the Google Play or Apple App Store.
Disclaimer: This site is designed to offer information for general educational purposes only. The health information furnished on this site and the interactive responses are not intended to be professional advice and are not intended to replace personal consultation with a qualified physician, pharmacist, or other healthcare professional. We cannot provide individual medical advice. You must always seek the advice of a professional for questions related to a disease, disease symptoms, and appropriate therapeutic treatments.
About Batten's disease (Neuronal ceroid lipofuscinosis)
About Neuronal ceroid lipofuscinosis